Archive for the ‘Crispr’ Category

For Immediate Release

Chicago, IL February 11, 2021 Zacks Equity Research Shares of Meridian Bioscience, Inc. VIVO as the Bull of the Day, CRISPR Therapeutics AG CRSP as the Bear of the Day. In addition, Zacks Equity Research provides analysis on Tilray, Inc. TLRY and Uber Technologies, Inc. UBER.

Here is a synopsis of all four stocks:

Meridian Bioscienceis a $1 billion provider of diagnostic test kits for gastrointestinal and respiratory infectious diseases. The company is expected to grow sales 32% this year to $335 million.

And after reporting a strong beat-and-raise quarter last week, analysts had to boost their EPS estimates over 30% from $1.24 to $1.63. So VIVO is back to a Zacks #1 Rank, sporting a projected 52% rise in profits.

I wrote about VIVO in early January and said it wasn't too late for investors to grab hold of this profit rocket near $20 per share...

"Bottom line on VIVO: I always pay attention to small companies growing their sales rapidly as they could become acquisition targets by larger biopharma or MedTech players. Buying VIVO near $20 offers excellent risk/reward, with or without an M&A suitor."

And then last week, right before the company report, I produced a video and article where I talked about why the COVID-19 testing stocks were under-appreciated given their fantastic growth...

Biotech Bonanza: COVID Launches Science at Warp Speed

Well here we are as VIVO pushed to 13-year highs above $30 with the earnings surprise and strong upside guidance.

Following the quarterly report, Piper Sandler analyst Steven Mah, a consistently bullish VIVO fan, raised his price target on Meridian Bioscience to $34 from $26 and reiterated an Overweight rating. Mah believes the company guidance was once again "very conservative" citing management's "pragmatic approach given the limited visibility on the durability of COVID-19 tailwinds."

Mah explained his increased confidence in Meridian's longer-term COVID-19 tailwinds given the Biden Administration's testing stance, slower than expected vaccine rollout, and new strain emergence.

Story continues

Meridian Bioscience develops, manufactures, distributes, and sells diagnostic test kits primarily for gastrointestinal and respiratory infectious diseases, and elevated blood lead levels worldwide. The company operates through Diagnostics and Life Science segments. They describe their mission as helping providers make better diagnostic decisions with a focus on gastrointestinal, neonatal, pediatrics, and respiratory conditions.

The Diagnostics segment offers testing platforms, including real-time PCR (polymerase chain reaction) amplification under the Revogene brand; isothermal DNA amplification under the Alethia brand; lateral flow immunoassay using fluorescent chemistry under the Curian brand; rapid immunoassay under the ImmunoCard and ImmunoCard STAT! brands; enzyme-linked immunoassays under the PREMIER brand; anodic stripping voltammetry under the LeadCare and PediaStat brands; and urea breath testing for H. pylori under the BreathID brand.

I have written often in the past few months of specialized diagnostic companies likeQuidelandHologicas they build new revenue streams from SARS-CoV-2 testing. These revenue streams are likely sustainable as the virus mutates and requires modified tests.

And I recently bought shares of VIVO for the Zacks Healthcare Innovators portfolio because I liked the growth outlook for this small player in a rapidly expanding market for rapid diagnostics -- including coronavirus testing which will continue to be part of our lives for years to come, even with vaccines.

While Meridian Bioscience is a David among diagnostic Goliaths, its long and fascinating history surprised me. From the company website...

In 1977, Bill Motto founded Meridian Bioscience on a $500 investment in his Cincinnati homes basement. Meridians first product was distributing a rapid fungal test developed by the University of Kentucky. While calling on his hospital and research customers, Bill noticed there was no easy, clean way to transport patient samples. He developed the innovative Para-Pak stool transport system to meet this need.

As the product line grew, so did Meridians research and development, leading to a breakthrough in 1982 with a 10-minute rapid test for strep throat. Before the Meridian test, doctors would have to wait for two to three days for a culture result. Innovation continued as the company brought several cutting edge diagnostic technologies to market, including a DNA testing platform and first-of-their-kind tests for C. difficile, E. coli, H. pylori amongst others.

The new bottom line on VIVO:I continue to hold the shares and would recommend new positions between $25 and $27 looking for new bull market highs above $30 by June.

Disclosure: I own shares of QDEL, HOLX, and VIVO for the Zacks Healthcare Innovators portfolio.

CRISPR Therapeuticsis one of my favorite biotech companies as the big leader among gene-editing pioneers.

But I had to let the stock go -- right before shares launched into the December ASH meeting (American Society of Hematology) -- because analysts were so bearish on the outlook for when the R&D pipeline would produce any revenues, much less profits.

Of course, talking about revenues and profits for world-changing, early-stage medical science is almost always a non sequitur.

Still in late summer, I let go of my CRSP shares for a 71% gain. But as I describe in this September video and article, it was not the first, nor the last, of great trading gains in the greatest of CRISPR companies...

CRISPR Stocks: Buy or Trade?

So why did CRSP launch from $110 to $210 in December and January?

It was mostly about investors recognizing that the company's early data in treating debilitating illnesses like Sickle Cell Disease (SCD) could indeed become world-changing for millions afflicted with the genetic impairment to their red blood cells.

SCD comprises a group of disorders that cause red blood cells to become misshapen and break down. Red blood cells contort into a sickle shape, and die early, leaving a shortage of healthy red blood cells (sickle cell anemia), and can block blood flow causing pain (sickle cell crisis). Infections, pain, and fatigue are symptoms of sickle cell disease. Current treatments include frequent medications, blood transfusions and, in extreme cases, a bone-marrow transplant -- but no cures.

As Antonio Regalado wrote in the MIT Technology Review wrote last week, "The burden of sickle-cell, an inherited disease that shortens lives by decades (or, in poor regions, kills during childhood), falls most heavily on Black people in equatorial Africa, Brazil, and the US. HIV has also become a lingering scourge: about two-thirds of people living with the virus, or dying from it, are in Africa."

I explained some of this potential in this vlog on December 10...

CRISPR Gene Editing: Owning the Future of Medicine

A secondary "igniter" of all CRISPR stocks launching higher in Dec-Jan (besides my video commentaries linked above) was the investment activity of Cathie Wood and her revolutionary ETF firm ARK Invest.

I produced a video and article about her one-woman investor revolution in early January with her monster ETFsARK Innovation:

How Cathie Put the Wood to Wall Street: TSLA, SQ, ROKU, CRSP, BIDU

There will be a time to buy CRSP again. But as with all emerging Biotechs, you sometimes have to wait for the next clinical data catalyst -- or an M&A one.

I'm betting the latter is the sooner driver of the next move from a $10 billion market cap to a $20 billion one.

Stay CRSPy!

Cooker

Kevin Cook is a Senior Stock Strategist for Zacks Investment Research where he runs the Healthcare Innovators portfolio.

Markets continued to hover close to the zero-line as of Wednesdays close, with just the Dow finishing in the green among major indexes. The blue-chips rose 0.20% for a new all-time high, while the Nasdaq, S&P 500 and Russell 2000 all took a breather: -0.25%, -0.03% and -0.72% on the day.

One of the major pot stocks we discussed yesterday in this space,Tilray, continues its big run in what looks more like the latest short-squeeze stock, with Reddit groups now piling into the marijuana-based pharmaceutical company of late. Shares shot up another 51% Wednesday, following a +44% performance yesterday; Tilray is now up 400% in just the last month alone.

For sure, increased acceptance in U.S. states and countries around the world are a reason for the stock to do well. However, its market cap has more than doubled and the company has no P/E because it is forecast for negative earnings both in the upcoming quarterly report and full fiscal year. Tilray is up another 10% in late trading, up near $71 per share. This stock was trading at $19 per share on February 1st.

Uber followed a nice 6% bump in regular-day Wednesday trading with a worse-than-expected fiscal Q3 report, missing on the bottom line by a penny to -54 cents per share (though still better than the -64 cents the company reported in the year-ago quarter). Revenues grew $3.17 billion in the quarter, well off the expected pace of $3.55 billion in the Zacks consensus. Yet shares are only selling off minimally in the after-market; the company still says its on track to its profitability goal in 2021.

Though Uber posted a net loss per year of $6.8 billion on ride-sharing revenues down 52% year over year, its Uber Eats delivery service grew 224% in its fiscal Q3. Monthly active platform consumers gained a million more than predicted in the quarter, +93 million. And when one figures in the ride-sharing comeback seemingly inevitable as the Covid-19 pandemic is finally beaten back with vaccinations, we see that Uber looks to have weathered its worst-possible storm and survived.For more on UBER's earnings, click here.

Speaking of the coronavirus, nearly 45 million vaccination doses have now been administered, and the post-holiday season peak now looks to have been successfully scaled. We are now back to 7-day case rates back where they were in October and, importantly, pointed in the right direction. More than 27 million Americans have reportedly contracted Covid-19, leading to more than 466K fatalities. More good news: the death rate is now flat for those whove gotten the worst of the disease.

Questions or comments about this article and/or its author? Click here>>

Experts extracted 7 stocks from the list of 220 Zacks Rank #1 Strong Buys that have beaten the market more than 2X over with a stunning average gain of +24.9% per year.

These 7 were selected because of their superior potential for immediate breakout.

See these time-sensitive tickers now >>

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Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free reportMeridian Bioscience Inc. (VIVO) : Free Stock Analysis ReportCRISPR Therapeutics AG (CRSP) : Free Stock Analysis ReportTo read this article on Zacks.com click here.

Read more:
Meridian Bioscience, CRISPR Therapeutics, Tilray and Uber highlighted as Zacks Bull and Bear of the Day - Yahoo Finance

The Global CRISPR Technology Market was valued at USD 696.7 Million in 2020 and is forecasted to reach USD 3.94 Bn at a CAGR of +24% by 2028.

CRISPR is a technology that can be used to edit genes and, as such, will likely change the world. The essence of CRISPR is simple: its a way of finding a specific bit of DNA inside a cell. After that, the next step in CRISPR gene editing is usually to alter that piece of DNA.

CRISPR-Cas9 was adapted from a naturally occurring genome editing system in bacteria. The bacteria capture snippets of DNA from invading viruses and use them to create DNA segments known as CRISPR arrays. The CRISPR arrays allow the bacteria to remember the viruses (or closely related ones).

With CRISPR, scientists can create a short RNA template in just a few days using free software and a DNA starter kit that costs $65 plus shipping. Unlike protein-based technologies, the RNA in CRISPR can be reprogrammed to target multiple genes.

The company has a healthy balance sheet with $1.4 billion in cash and very little debt. CRISPR has the financial flexibility to fund its research programs for many more years to come. So it can prove to be an attractive investment for healthcare investors in the next five years.

Request for a sample report here @ https://www.reportconsultant.com/request_sample.php?id=43967

Major Players Covered in this Report:

CRISPR Technology Market Study assures you to advise higher than your competition. With Structured tables and figures examining the CRISPR Technology, the research document provides you a leading product, submarkets, revenue size and forecast to 2028.

The study report offers a comprehensive analysis of CRISPR Technology market size across the globe as regional and country level market size analysis, CAGR estimation of market growth during the forecast period, revenue, key drivers, competitive background and sales analysis of the payers. Along with that, the report explains the major challenges and risks to face in the forecast period.

The research report of the CRISPR Technology market offers broad analysis about the industry on the basis of different key segments. Moreover, the research report presents a comprehensive analysis about the opportunities, new products, and technological innovations in the market for the players.

Additionally, the research report on CRISPR Technology market provides an in depth analysis about market status, market size, revenue share, industry development trends, products advantages and disadvantages of the enterprise, enterprise competition pattern, industrial policy and regional industrial layout characteristics. Thus the study report offers a comprehensive analysis of market size across the globe as regional and country level market size analysis, estimation of market growth during the forecast period.

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Market segmentation by Product Type:

Market segmentation by Applications:

Market segmentation by regions:

This study also covers company profiling, specifications and product picture, sales, market share and contact information of various regional, international and local vendors of Global CRISPR Technology Market. The market opposition is frequently developing greater with the rise in scientific innovation and M&A activities in the industry. Additionally, many local and regional vendors are offering specific application products for varied end-users. The new merchant applicants in the market are finding it hard to compete with the international vendors based on reliability, quality and modernism in technology.

Detailed TOC of CRISPR Technology Market Research Report-

CRISPR Technology Introduction and Market Overview

CRISPR Technology Market, by Application

CRISPR Technology Industry Chain Analysis

CRISPR Technology Market, by Type

Industry Manufacture, Consumption, Export, Import by Regions

Industry Value ($) by Region

CRISPR Technology Market Status and SWOT Analysis by Regions

Major Region of CRISPR Technology Market

Major Companies List

Conclusion

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Our research reports will give you the most realistic and incomparable experience of revolutionary market solutions. We have effectively steered business all over the world through our market research reports with our predictive nature and are exceptionally positioned to lead digital transformations. Thus, we craft greater value for clients by presenting progressive opportunities in the global futuristic market.

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CRISPR Technology Market hits at $3.94 Bn by 2028 with Thermo Fisher Scientific, Merck KGaA, GenScript, Integrated DNA Technologies (IDT) - The...

California headquartered, Caribou, isa clinical-stage CRISPR genome editing biotechnology company.

The collaboration leverages Caribous next-generation CRISPR genome editing technology platform and AbbVies antigen-specific binders. AbbVie will utilize Caribous next-generation Cas12a CRISPR hybrid RNA-DNA (chRDNA) genome editing and cell therapy technologies to research and develop two new CAR-T cell therapies.

Under the terms of deal, Caribou will receive US$40m in an upfront cash payment and equity investment and up to US$300m in future development, regulatory, and launch milestones.

Although allogeneic CAR-T cell therapies have shown early promise in some cancer patients, the need for overcoming the rejection of allogeneic CAR-T cells by the host immune system remains a key challenge to their broader development, said the companies.

Employing Caribous CRISPR genome editing platform to engineer CAR-T cells to withstand host immune attack would enable the development of the next-generation cellular therapies to benefit a broader patient population, claim the partners.

Caribou will conduct certain pre-clinical research, development, and manufacturing activities for the collaboration programs, and AbbVie will reimburse Caribou for all such activities pursuant to the collaboration, while AbbVie will be responsible for all clinical development, commercialization, and manufacturing efforts.

AbbVie also has the option to pay a fee to expand the collaboration to include up to an additional two CAR-T cell therapies.

We are excited to partner with AbbVie on the development of new CAR-T cell therapies. This collaboration validates Caribous differentiated next-generation CRISPR genome editing technologies that provide best-in-class efficiency and specificity, said Rachel Haurwitz, CEO of Caribou.

CAR-T therapies have shown to be a promising breakthrough in cancer treatment, Collaborating with Caribou and their cutting-edge CRISPR platform will help AbbVie advance our efforts to deliver new hope for patients, said Steve Davidsen, VP, oncology discovery, AbbVie.

Outside of this collaboration, Caribou is advancing an internal pipeline of allogeneic cell therapies for oncology. CB-010, its lead allogeneic CAR-T cell program, targets CD19 and is being evaluated in a Phase 1 clinical trial for patients with relapsed/refractory B cell non-Hodgkin lymphoma.

CB-011, Caribous second allogeneic CAR-T cell therapy, targets BCMA for multiple myeloma, while CB-012, Caribous third allogeneic CAR-T cell therapy, targets CD371 for acute myeloid leukemia.

CB-011 and CB-012 are in preclinical development.

Additionally, Caribou is developing iPSC-derived allogeneic natural killer (NK) cell therapies for solid tumors.

See the original post:
AbbVie and Caribou partner on CAR-T cell therapy development - BioPharma-Reporter.com

Due to the unique consequences of the pandemic, we are able to catch a glimpse of a potential future. One where we sit, plugged into our computers, devoid of physical human connection. What will society look like after the pandemic? Will we continue to stay isolated? Surely advancements in technology have played key roles in prolonging our lives, allowing us to continue to work and socialise, but to what end? With these newly emerging technologies are we destined to live forever, in a suspended state, in front of the glow of our 4k computer screens? Will gene editing technologies be used to keep us alive forever so that we can binge watch infinite Netflix shows, send meaningless emails and scroll through social media feeds?

Q4 2020 hedge fund letters, conferences and more

The Institute of Zoology of the China Academy of Science has successfully prolonged the lives of mice by using CRISPR/Cas 9. CRISPR has become a relatively simple and popular way to edit strands of DNA. The CRISPR/Cas 9 study found a gene tied to cellular senescence (which tells cells to stop growing) and also, that CRISPR/Cas9 treatment can make partially dormant the aging process. CRISPR/Cas9 treatment allowed mice to live 25% longer and be physically stronger. Biologists see these results being relatively easy to reproduce on humans in a clinical setting.

Company managements looking to achieve earnings growth often default to cost cutting, stock buyback, accounting gimmicks and other methods. But there is another way. More often than not, managements overlook pricing as a driver of earnings growth. Pricing power can be an effect way of boosting a company's bottom line. Read More

Existing in a world where individuals are able to receive treatment to live longer borders dystopian science fiction. The treatment can reduce the need for medical attention by potentially reducing injuries, heart attacks, and organ failures.

In part, due to breakthroughs in the tech and science industries, life expectancy in the 21st century is projected to steadily increase. In a study published in the Lancet, average life expectancy is predicted to rise in 35 industrialized countries by 4.4 years in men and women by 2030.

Life expectancy will likely increase as we migrate away from laborer positions. Currently, the National Center for Health Statistics puts unintentional accidents, primarily happening within labor positions, as the third leading cause of death in the U.S. Many industrialized countries, like the U.S., have been witness to a slow disappearance of the labor class, but the pandemic has made that increasingly more apparent. Businesses are shifting towards automated technology to replace physical human interactions to curb the spread of Covid-19. Even within agriculture and farming industries, already abundant with machine automation, companies are pushing even further away from human labor in an effort to reduce virus rates.

Simultaneously, we are witness to the emergence of new and remote jobs and work settings. Homes are new sites for schooling, work and entertainment. Before the pandemic there was already a struggle to maintain a separation between home and work identities. The pandemic has exacerbated this problem. How do we find rest and recuperation when we are living within the office space? Time on the job stretches on forever as we receive work emails while watching Netflix with our families. If life expectancy in humans gets extended by using CRISPR/Cas 9 are we just creating our own version of purgatory? Is our future one where our time working stretches on seemingly infinitely while we simultaneously cease to age? Are we becoming the perfect machine, one that is held together by technological advancements that inadvertently disembody and dehumanise us?

Was society slowly transitioning toward isolation before the pandemic? Is isolation a byproduct of neoliberalism? Gated communities, mass incarceration, office cubicles, segregation in neighborhoods, retirement homes, hospitals, national borders and private properties all verify how neoliberalism operates. Now we are being asked to isolate within the confines of our homes. When the pandemic is over, will people continue to order food, work, shop and socialise from isolated and often virtual spaces or will we be able to shift back into the more public and physical? Will the last remaining physical laborers, those that are delivering goods to doorsteps, be replaced by driverless vehicles?

About the Author

Matthew Bacher is an author and professional artist and is working towards his MFA in Painting and Printmaking at San Diego State University. His work deals with postmodern ideas around nature, technology and the self. You can find his artwork atmbacherart.com.

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CRISPR Treatment Offers The Potential To Live Forever - ValueWalk

The term genome generally refers to the entire sequence of DNA of an organism. The genome includes genes: sequences of DNA with specific functions that are involved in the production of the proteins needed to carry out many biological roles. Genome editing is the deliberate alteration of a selected DNA sequence in a living cell. A strand of DNA is cut at a specific point and naturally existing cellular repair mechanisms, then fix the broken DNA strands.

The Australia Gene Editing market 2021 research provides a basic overview of the industry including definitions, classifications, applications and industry chain structure. The Australia Gene Editing market analysis is provided for the international markets including development trends, competitive landscape analysis, and key regions development status. Development policies and plans are discussed as well as manufacturing processes and cost structures are also analyzed. This report also states import/export consumption, supply and demand Figures, cost, price, revenue and gross margins.

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Top Vendors of Australia Gene Editing Market:-

CRISPR Therapeutics, Thermo Fisher Scientific, GenScript Corporation, Merck KgaA, Sangamo Therapeutics, Inc., Horizon Discovery Group, Integrated DNA Technologies, New England Biolabs, OriGene Technologies, Lonza Group, and Editas Medicine.

Australia Gene Editing Market, By Application

Australia Gene Editing Market, By Technology

Australia Gene Editing Market, By End-user:

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The study is a source of reliable data on:

-Key market segments and sub-segments

-Evolving market trends and dynamics

-Changing supply and demand scenarios

-Quantifying market opportunities through market sizing and market forecasting

-Tracking current trends/opportunities/challenges

-Competitive insights

-Opportunity mapping in terms of technological breakthroughs

The Australia Gene Editing market research report completely covers the vital statistics of the capacity, Development, value, cost/profit, supply/demand import/export, further divided by company and country, and by application/type for best possible updated data representation in the figures, tables, pie chart, and graphs.

Fundamentals of Table of Content:

About us:

Report Consultant A worldwide pacesetter in analytics, research and advisory that can assist you to renovate your business and modify your approach. With us, you will learn to take decisions intrepidly by taking calculative risks leading to lucrative businesses in the ever-changing market. We make sense of drawbacks, opportunities, circumstances, estimations and information using our experienced skills and verified methodologies.

Our research reports will give you the most realistic and incomparable experience of revolutionary market solutions. We have effectively steered businesses all over the world through our market research reports with our predictive nature and are exceptionally positioned to lead digital transformations. Thus, we craft greater value for clients by presenting progressive opportunities in the futuristic market.

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Australia Gene Editing Market to Garner Astonishing CAGR by 2028 with Top Key Players: CRISPR Therapeutics, Thermo Fisher Scientific, GenScript...

Chinese scientists have successfully prolonged the lives of mice by using CRISPR.

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February10, 20214 min read

Due to the unique consequences of the pandemic, we are able to catch a glimpse of a potential future. One where we sit, plugged into our computers, devoid of physical human connection. What will society look like after thepandemic? Will we continue to stay isolated? Surely advancements in technology have played key roles in prolonging our lives, allowing us to continue to work and socialise, but to what end? With these newly emerging technologies are we destined to live forever, in a suspended state, in front of the glow of our 4k computer screens? Will gene editing technologies be used to keep us alive forever so that we can binge watch infinite Netflix shows, send meaningless emails and scroll through social media feeds?

The Institute of Zoology of the China Academy of Science has successfully prolonged the lives of mice by using CRISPR/Cas 9. CRISPR has become a relatively simple and popular way to edit strands of DNA. The CRISPR/Cas 9 study found a gene tied to cellular senescence (which tells cells to stop growing) and also, that CRISPR/Cas9 treatment can make partially dormant theagingprocess. CRISPR/Cas9 treatment allowed mice to live 25% longer and be physically stronger. Biologists see these results being relatively easy to reproduce on humans in a clinical setting.

Existing in a world where individuals are able to receive treatment to live longer borders dystopian science fiction. The treatment can reduce the need for medical attention by potentially reducing injuries, heart attacks, and organ failures.

In part, due to breakthroughs in the tech and science industries, life expectancy in the 21st century is projected to steadily increase. In a study published in the Lancet, average life expectancy is predicted to rise in 35 industrialized countries by 4.4 years in men and women by 2030.

Life expectancy will likely increase as we migrate away from laborer positions. Currently, the National Center for Health Statistics puts unintentional accidents, primarily happening within labor positions, as the third leading cause of death in the U.S. Many industrialized countries, like the U.S., have been witness to a slow disappearance of the labor class, but the pandemic has made that increasingly more apparent. Businesses are shifting towards automated technology to replace physical human interactions to curb the spread of Covid-19. Even within agriculture andfarmingindustries, already abundant with machine automation, companies are pushing even further away from human labor in an effort to reduce virus rates.

Simultaneously, we are witness to the emergence of new and remote jobs and work settings.Homesare new sites for schooling, work, andentertainment. Before the pandemic, there was already a struggle to maintain a separation between home and work identities. The pandemic has exacerbated this problem. How do we find rest and recuperation when we are living within the office space? Time on the job stretches on forever as we receive work emails while watching Netflix with our families. If life expectancy in humans gets extended by using CRISPR/Cas 9 are we just creating our own version of purgatory? Is our future one where our time working stretches on seemingly infinitely while we simultaneously cease to age? Are we becoming the perfect machine, one that is held together by technological advancements that inadvertently disembody and dehumanize us?

Was society slowly transitioning toward isolation before the pandemic? Is isolation a byproduct of neoliberalism? Gated communities, mass incarceration, office cubicles, segregation in neighborhoods,retirementhomes, hospitals, national borders and private properties all verify how neoliberalism operates. Now we are being asked to isolate ourselves within the confines of our homes. When the pandemic is over, will people continue to order food, work, shop, and socialize from isolated and often virtual spaces or will we be able to shift back into the more public and physical? Will the last remaining physical laborers, those that are delivering goods to doorsteps, be replaced by driverless vehicles?

Here is the original post:
CRISPR Offers the Potential to Live Forever, But to What End? - Entrepreneur

TipRanks

Lets talk about growth. With corona receding, politics growing less exciting, and a new year ahead, investors are getting optimistic and that means theres a hunt for stocks that will bring in strong returns. In other words, growth stocks. In a recent interview, Jan Hatzius, chief economist at investment giant Goldman Sachs, said that he sees GDP growth in 2Q21 hitting as high as 10%. In an environment like that, most stocks are going to show a growth trend. Now, we all know that past performance wont guarantee future results. Still, the best place to start looking for tomorrows high-growth stocks is among yesterdays winners. Bearing this in mind, we set out to find stocks flagged as exciting growth plays by Wall Street. Using TipRanks database, we locked in on three analyst-backed names that have already notched impressive gains and boast solid growth narratives for the long-term. Kaleyra (KLR) We will start with Kaleyra, a cloud computing company offering communications solutions. The companys SaaS platform supports SMS, voice calls, and chatbots a product with obvious applications and value in todays office climate, with the strong push to telecommuting and remote work. Kaleyra boasts over 3,500 customers, who make 3 billion voice calls and sent 27 billion text messages in 2019 (the last year with full numbers available). Over the past 6 months, KLR shares have shown tremendous growth, appreciating 155%. Kaleyras revenues have grown along with the share value. The companys 3Q20 results hit $38.3 million, the best since KLR went public. While Kaleyra still runs a net earnings loss each quarter, the Q3 EPS was the lowest such loss in the past four quarters. Maxim analyst Allen Klee is bullish on KLR, seeing recent growth and product offerings as indicative of future performance. Over the past few years, Kaleyra has posted double-digit revenue growth and positive adjusted EBITDA. We forecast revenue growth of 9%, 22%, and 28% for 2020-2022. We project adjusted EBITDA declines in 2020 to reflect public company costs and COVID-19, but growth at over twice the rate of revenue for the following two years. We expect benefits from operating leverage, low-cost tech employees, cost volume discounts as the company expands, and margin improvement from new offerings and geographies. Over the longer term, we believe the company can grow revenue close to 30% with even faster bottom line growth," Klee opined. With such growth, its no wonder Klee takes a bullish stance on KLR. To kick off his coverage, the analyst published a Buy rating and set a $22 price target. This figure implies a 45% for the coming year. (To watch Klees track record, click here) Overall, based on the 3 Buy ratings vs no Holds or Sells assigned in the last three months, Wall Street analysts agree that this Strong Buy is a solid bet. It also doesnt hurt that its $19 average price target implies ~26% upside potential. (See KLR stock analysis on TipRanks) Vista Outdoor (VSTO) Next up, Vista Outdoor, is a venerable company that saw its niche gain attractiveness in recent times. Vista is a sporting goods company, with 40 brands in two main divisions: outdoor products and shooting sports. Vistas brands include well-known names as Bushnell Golf, CamelBak, and Remington. The company has found a burst of success in the corona year as people have turned more and more to outdoor activities that can be practiced solo or in small groups expanding the customer base. VSTO shares are up as a result, by 214% in the last 12 months. Vistas earnings reflect the increase in consumer interest in outdoor sports. The companys EPS grew in 2020, turning from a net loss to a $1.34 per share profit in the fiscal Q2 report (released in November). The fiscal Q3 report, released earlier this month, showed lower earnings, at $1.31 per share, but was still considered solid by the company, as it covered winter months when the company normally sees a revenue decline. Both quarters showed strong year-over-year EPS gains. Covering Vista for B. Riley, 5-star analyst Eric Wold sees several avenues for continued growth by Vista. He is impressed by the growth in firearm and ammunition sales, and by the price increase for products in both the outdoor goods and the shooting sports divisions. Given our expectation that the increased industry participation numbers for both outdoor products and shooting sports during the pandemic will represent an incremental tailwind for VSTO in the coming years beyond the impressive production visibility that has been created by depleted channel inventory levels, we continue to see an attractive set-up for baseline growth, Wold commented. Overall, Wold is bullish on the stock and rates it a Buy, with a $41 price target. This figure indicates room for 27% upside in the coming year. (To watch Wolds track record, click here) Vista is another company with a unanimous Strong Buy consensus rating. That rating is based on 9 recent reviews, all to Buy. VSTO shares have an average price target of $36.78, which gives an upside of 14% from the trading price of $32.15. (See VSTO stock analysis on TipRanks) Textainer Group Holdings (TGH) You might not think about the ubiquitous cargo container, but these deceptively simple metal boxes have changed the face of bulk transport since their breakout proliferation in the 1960s. These containers make it easy to organize, load, ship, and track vast amounts of cargo, and are especially valuable for their ease of switching; containers can be quickly loaded on or switched between ships, trains, and trucks. Textainer is a billion-dollar company that buys, owns, and leases shipping containers for the cargo industry. The company has over 250 customers, and boasts a fleet of 3 million twenty-foot equivalent units (TEUs). Textainer is also a major reseller of used containers, and operates from 500 depots around the world. Even during the corona pandemic, when international trading routes and patterns were badly disrupted, and the quarterly revenues were down year-over-year, Textainer saw share gains. The companys stock soared 110% over the past 12 months. The bulk of these gains have come in the past six months, as economies and trading patterns have begun to reopen. Looking at Textainer for B. Riley, analyst Daniel Day is deeply impressed. He sees this company as the lowest priced among its peer group, with a strong market share in a competitive industry. Day rates TGH a Buy, and his $31 price target suggests it has room for 57% growth ahead of it. In support of this bullish stance, Day writes, in part, We believe that TGH is an underfollowed, misunderstood name that is ideal for the portfolio of a deep value investor looking for cash flowgenerative names trading at a steep discount to intrinsic value. With new container prices at multiyear highs amid a resurgence in container shipping, we expect upcoming earnings results to be positive catalyst events for TGH Some stocks fly under the radar, and TGH is one of those. Day's is the only recent analyst review of this company, and it is decidedly positive. (See TGH stock analysis on TipRanks) To find good ideas for growth stocks trading at attractive valuations, visit TipRanks Best Stocks to Buy, a newly launched tool that unites all of TipRanks equity insights. Disclaimer: The opinions expressed in this article are solely those of the featured analysts. The content is intended to be used for informational purposes only. It is very important to do your own analysis before making any investment.

See the article here:
Global CRISPR Gene Editing Market (2020 to 2030) - Focus on Products, Applications, End-users, Country Data and Competitive Landscape -...

Dublin, Feb. 08, 2021 (GLOBE NEWSWIRE) -- The "Global CRISPR Gene Editing Market: Focus on Products, Applications, End Users, Country Data (16 Countries), and Competitive Landscape - Analysis and Forecast, 2020-2030" report has been added to ResearchAndMarkets.com's offering.

The global CRISPR gene editing market was valued at $846.2 million in 2019 and is expected to reach $10,825.1 million by 2030, registering a CAGR of 26.86% during the forecast.

The development of genome engineering with potential applications proved to reflect a remarkable impact on the future of the healthcare and life science industry. The high efficiency of the CRISPR-Cas9 system has been demonstrated in various studies for genome editing, which resulted in significant investments within the field of genome engineering. However, there are several limitations, which need consideration before clinical applications. Further, many researchers are working on the limitations of CRISPR gene editing technology for better results. The potential of CRISPR gene editing to alter the human genome and modify the disease conditions is incredible but exists with ethical and social concerns.

The growth is attributed to the increasing demand in the food industry for better products with improved quality and nutrient enrichment and the pharmaceutical industry for targeted treatment for various diseases. Further, the continued significant investments by healthcare companies to meet the industry demand and growing prominence for the gene therapy procedures with less turnaround time are the prominent factors propelling the growth of the global CRISPR gene editing market.

Research organizations, pharmaceutical and biotechnology industries, and institutes are looking for more efficient genome editing technologies to increase the specificity and cost-effectiveness, also to reduce turnaround time and human errors. Further, the evolution of genome editing technologies has enabled wide range of applications in various fields, such as industrial biotech and agricultural research. These advanced methods are simple, super-efficient, cost-effective, provide multiplexing, and high throughput capabilities. The increase in the geriatric population and increasing number of cancer cases, and genetic disorders across the globe are expected to translate into significantly higher demand for CRISPR gene editing market.

Furthermore, the companies are investing huge amounts in the research and development of CRISPR gene editing products, and gene therapies. The clinical trial landscape of various genetic and chronic diseases has been on the rise in recent years, and this will fuel the CRISPR gene editing market in the future.

Within the research report, the market is segmented based on product type, application, end-user, and region. Each of these segments covers the snapshot of the market over the projected years, the inclination of the market revenue, underlying patterns, and trends by using analytics on the primary and secondary data obtained.

Key Companies Profiled

Abcam, Inc., Applied StemCell, Inc., Agilent Technologies, Inc., Cellecta, Inc., CRISPR Therapeutics AG, Thermo Fisher Scientific, Inc., GeneCopoeia, Inc., GeneScript Biotech Corporation, Horizon Discovery Group PLC, Integrated DNA Technologies, Inc., Merck KGaA, New England Biolabs, Inc., Origene Technologies, Inc., Rockland Immunochemicals, Inc., Synthego Corporation, System Biosciences LLC, ToolGen, Inc., Takara Bio

Key Questions Answered in this Report:

Key Topics Covered:

1 Technology Definition

2 Research Scope

3 Research Methodology

4 Market Overview4.1 Introduction4.2 CRISPR Gene Editing Market Approach4.3 Milestones in CRISPR Gene Editing4.4 CRISPR Gene Editing: Delivery Systems4.5 CRISPR Technology: A Potential Tool for Gene Editing4.6 CRISPR Gene Editing Current Scenario4.7 CRISPR Gene Editing Market: Future Potential Application Areas

5 Global CRISPR Gene Editing Market, $Million, 2020-20305.1 Pipeline Analysis5.2 CRISPR Gene Editing Market and Growth Potential, 2020-20305.3 Impact of COVID-19 on CRISPR Gene Editing Market5.3.1 Impact of COVID-19 on Global CRISPR Gene Editing Market Growth Rate5.3.1. Impact on CRISPR Gene Editing Companies5.3.2 Clinical Trial Disruptions and Resumptions5.3.3 Application of CRISPR Gene Editing in COVID-19

6 Market Dynamics6.1 Impact Analysis6.2 Market Drivers6.2.1 Prevalence of Genetic Disorders and Use of Genome Editing6.2.2 Government and Private Funding6.2.3 Technology Advancement in CRISPR Gene Editing6.3 Market Restraints6.3.1 CRISPR Gene Editing: Off Target Effects and Delivery6.3.2 Ethical Concerns and Implications With Respect to Human Genome Editing6.4 Market Opportunities6.4.1 Expanding Gene and Cell Therapy Area6.4.2 CRISPR Gene Editing Scope in Agriculture

7 Industry Insights7.1 Introduction7.2 Funding Scenario7.3 Regulatory Scenario of CRISPR Gene Editing Market7.4 Pricing of CRISPR Gene Editing7.5 Reimbursement of CRISPR Gene Editing7.5.1 CRISPR Gene Editing: Insurance Coverage in the U.S.

8 CRISPR Gene Editing Patent Landscape8.1 Overview8.2 CRISPR Gene Editing Market Patent Landscape: By Application8.3 CRISPR Gene Editing Market Patent Landscape: By Region8.4 CRISPR Gene Editing Market Patent Landscape: By Year

9 Global CRISPR Gene Editing Market (by Product Type), $Million9.1 Overview9.2 CRISPR Products9.2.1 Kits and Enzymes9.2.1.1 Vector-Based Cas99.2.1.2 DNA-Free Cas99.2.2 Libraries9.2.3 Design Tools9.2.4 Antibodies9.2.5 Other Products9.3 CRISPR Services9.3.1 gRNA Design and Vector Construction9.3.2 Cell Line and Engineering9.3.3 Screening Services9.3.4 Other Services

10 CRISPR Gene Editing Market (by Application), $Million10.1 Overview10.2 Agriculture10.3 Biomedical10.3.1 Gene Therapy10.3.2 Drug Discovery10.3.3 Diagnostics10.4 Industrial10.5 Other Applications

11 Global CRISPR Gene Editing Market (by End User)11.1 Academic Institutions and Research Centers11.2 Biotechnology Companies11.3 Contract Research Organizations (CROs)11.4 Pharmaceutical and Biopharmaceutical Companies

12 Global CRISPR Gene Editing Market (by Region)12.1 Introduction12.2 North America12.3 Europe12.4 Asia-Pacific12.5 Latin America

13 Competitive Landscape13.1 Key Developments and Strategies13.1.1 Overview13.1.1.1 Regulatory and Legal Developments13.1.1.2 Synergistic Activities13.1.1.3 M&A Activities13.1.1.4 Funding Activities13.2 Market Share Analysis13.3 Growth Share Analysis

14 Company Profiles14.1 Overview14.2 Abcam, Inc.14.2.1 Company Overview14.2.2 Role of Abcam, Inc. in the Global CRISPR Gene Editing Market14.2.3 Financials14.2.4 SWOT Analysis14.3 Applied StemCell, Inc.14.3.1 Company Overview14.3.2 Role of Applied StemCell, Inc. in the Global CRISPR Gene Editing Market14.3.3 SWOT Analysis14.4 Agilent Technologies, Inc.14.4.1 Company Overview14.4.2 Role of Agilent Technologies, Inc. in the Global CRISPR Gene Editing Market14.4.3 Financials14.4.4 R&D Expenditure, 2017-201914.4.5 SWOT Analysis14.5 Cellecta, Inc.14.5.1 Company Overview14.5.2 Role of Cellecta, Inc. in the Global CRISPR Gene Editing Market14.5.3 SWOT Analysis14.6 CRISPR Therapeutics AG14.6.1 Company Overview14.6.2 Role of CRISPR Therapeutics AG in the Global CRISPR Gene Editing Market14.6.3 Financials14.6.4 R&D Expenditure, 2017-201914.6.5 SWOT Analysis14.7 Thermo Fisher Scientific, Inc. INC14.7.1 Company Overview14.7.2 Role of Thermo Fisher Scientific, Inc. in the Global CRISPR Gene Editing Market14.7.3 Financials14.7.4 R&D Expenditure, 2017-201914.7.5 SWOT Analysis14.8 GeneCopoeia, Inc.14.8.1 Company Overview14.8.2 Role of GeneCopoeia, Inc. in the Global CRISPR Gene Editing Market14.8.3 SWOT Analysis14.9 GeneScript Biotech Corporation14.9.1 Company Overview14.9.2 Role of GenScript Biotech in the Global CRISPR Gene Editing Market14.9.3 Financials14.9.4 SWOT Analysis14.1 Horizon Discovery Group PLC14.10.1 Company Overview14.10.2 Role of Horizon Discovery Group PLC in the Global CRISPR Gene Editing Market14.10.3 Financials14.10.4 SWOT Analysis14.11 Integrated DNA Technologies, Inc.14.11.1 Company Overview14.11.2 Role of Integrated DNA Technologies, Inc. in the Global CRISPR Gene Editing Market14.11.3 SWOT Analysis14.12 Merck KGaA14.12.1 Company Overview14.12.2 Role of Merck KGaA in the Global CRISPR Gene Editing Market14.12.3 Financials14.12.4 SWOT Analysis14.13 New England Biolabs, Inc.14.13.1 Company Overview14.13.2 Role of Integrated DNA Technologies, Inc. in the Global CRISPR Gene Editing Market14.13.3 SWOT Analysis14.14 Origene Technologies, Inc.14.14.1 Company Overview14.14.2 Role of Origene Technologies, Inc. in the Global CRISPR Gene Editing Market14.14.3 SWOT Analysis14.15 Rockland Immunochemicals, Inc.14.15.1 Company Overview14.15.2 Role of Rockland Immunochemicals, Inc. in the Global CRISPR Gene Editing Market14.15.3 SWOT Analysis14.16 Synthego Corporation14.16.1 Company Overview14.16.2 Role of Synthego Corporation in the Global CRISPR Gene Editing Market14.16.3 SWOT Analysis14.17 System Biosciences LLC14.17.1 Company Overview14.17.2 Role of System Biosciences LLC in the Global CRISPR Gene Editing Market14.17.3 SWOT Analysis14.18 ToolGen, Inc.14.18.1 Company Overview14.18.2 Role of ToolGen, Inc. in the Global CRISPR Gene Editing Market14.18.3 SWOT Analysis14.19 Takara Bio14.19.1 Company Overview14.19.2 Role of Takara Bio in the Global CRISPR Gene Editing Market14.19.3 Financials14.19.4 SWOT Analysis

For more information about this report visit https://www.researchandmarkets.com/r/c7om7t

See more here:
Outlook on the CRISPR Gene Editing Global Market to 2030 - Analysis and Forecasts - GlobeNewswire

TipRanks

Lets talk about growth. With corona receding, politics growing less exciting, and a new year ahead, investors are getting optimistic and that means theres a hunt for stocks that will bring in strong returns. In other words, growth stocks. In a recent interview, Jan Hatzius, chief economist at investment giant Goldman Sachs, said that he sees GDP growth in 2Q21 hitting as high as 10%. In an environment like that, most stocks are going to show a growth trend. Now, we all know that past performance wont guarantee future results. Still, the best place to start looking for tomorrows high-growth stocks is among yesterdays winners. Bearing this in mind, we set out to find stocks flagged as exciting growth plays by Wall Street. Using TipRanks database, we locked in on three analyst-backed names that have already notched impressive gains and boast solid growth narratives for the long-term. Kaleyra (KLR) We will start with Kaleyra, a cloud computing company offering communications solutions. The companys SaaS platform supports SMS, voice calls, and chatbots a product with obvious applications and value in todays office climate, with the strong push to telecommuting and remote work. Kaleyra boasts over 3,500 customers, who make 3 billion voice calls and sent 27 billion text messages in 2019 (the last year with full numbers available). Over the past 6 months, KLR shares have shown tremendous growth, appreciating 155%. Kaleyras revenues have grown along with the share value. The companys 3Q20 results hit $38.3 million, the best since KLR went public. While Kaleyra still runs a net earnings loss each quarter, the Q3 EPS was the lowest such loss in the past four quarters. Maxim analyst Allen Klee is bullish on KLR, seeing recent growth and product offerings as indicative of future performance. Over the past few years, Kaleyra has posted double-digit revenue growth and positive adjusted EBITDA. We forecast revenue growth of 9%, 22%, and 28% for 2020-2022. We project adjusted EBITDA declines in 2020 to reflect public company costs and COVID-19, but growth at over twice the rate of revenue for the following two years. We expect benefits from operating leverage, low-cost tech employees, cost volume discounts as the company expands, and margin improvement from new offerings and geographies. Over the longer term, we believe the company can grow revenue close to 30% with even faster bottom line growth," Klee opined. With such growth, its no wonder Klee takes a bullish stance on KLR. To kick off his coverage, the analyst published a Buy rating and set a $22 price target. This figure implies a 45% for the coming year. (To watch Klees track record, click here) Overall, based on the 3 Buy ratings vs no Holds or Sells assigned in the last three months, Wall Street analysts agree that this Strong Buy is a solid bet. It also doesnt hurt that its $19 average price target implies ~26% upside potential. (See KLR stock analysis on TipRanks) Vista Outdoor (VSTO) Next up, Vista Outdoor, is a venerable company that saw its niche gain attractiveness in recent times. Vista is a sporting goods company, with 40 brands in two main divisions: outdoor products and shooting sports. Vistas brands include well-known names as Bushnell Golf, CamelBak, and Remington. The company has found a burst of success in the corona year as people have turned more and more to outdoor activities that can be practiced solo or in small groups expanding the customer base. VSTO shares are up as a result, by 214% in the last 12 months. Vistas earnings reflect the increase in consumer interest in outdoor sports. The companys EPS grew in 2020, turning from a net loss to a $1.34 per share profit in the fiscal Q2 report (released in November). The fiscal Q3 report, released earlier this month, showed lower earnings, at $1.31 per share, but was still considered solid by the company, as it covered winter months when the company normally sees a revenue decline. Both quarters showed strong year-over-year EPS gains. Covering Vista for B. Riley, 5-star analyst Eric Wold sees several avenues for continued growth by Vista. He is impressed by the growth in firearm and ammunition sales, and by the price increase for products in both the outdoor goods and the shooting sports divisions. Given our expectation that the increased industry participation numbers for both outdoor products and shooting sports during the pandemic will represent an incremental tailwind for VSTO in the coming years beyond the impressive production visibility that has been created by depleted channel inventory levels, we continue to see an attractive set-up for baseline growth, Wold commented. Overall, Wold is bullish on the stock and rates it a Buy, with a $41 price target. This figure indicates room for 27% upside in the coming year. (To watch Wolds track record, click here) Vista is another company with a unanimous Strong Buy consensus rating. That rating is based on 9 recent reviews, all to Buy. VSTO shares have an average price target of $36.78, which gives an upside of 14% from the trading price of $32.15. (See VSTO stock analysis on TipRanks) Textainer Group Holdings (TGH) You might not think about the ubiquitous cargo container, but these deceptively simple metal boxes have changed the face of bulk transport since their breakout proliferation in the 1960s. These containers make it easy to organize, load, ship, and track vast amounts of cargo, and are especially valuable for their ease of switching; containers can be quickly loaded on or switched between ships, trains, and trucks. Textainer is a billion-dollar company that buys, owns, and leases shipping containers for the cargo industry. The company has over 250 customers, and boasts a fleet of 3 million twenty-foot equivalent units (TEUs). Textainer is also a major reseller of used containers, and operates from 500 depots around the world. Even during the corona pandemic, when international trading routes and patterns were badly disrupted, and the quarterly revenues were down year-over-year, Textainer saw share gains. The companys stock soared 110% over the past 12 months. The bulk of these gains have come in the past six months, as economies and trading patterns have begun to reopen. Looking at Textainer for B. Riley, analyst Daniel Day is deeply impressed. He sees this company as the lowest priced among its peer group, with a strong market share in a competitive industry. Day rates TGH a Buy, and his $31 price target suggests it has room for 57% growth ahead of it. In support of this bullish stance, Day writes, in part, We believe that TGH is an underfollowed, misunderstood name that is ideal for the portfolio of a deep value investor looking for cash flowgenerative names trading at a steep discount to intrinsic value. With new container prices at multiyear highs amid a resurgence in container shipping, we expect upcoming earnings results to be positive catalyst events for TGH Some stocks fly under the radar, and TGH is one of those. Day's is the only recent analyst review of this company, and it is decidedly positive. (See TGH stock analysis on TipRanks) To find good ideas for growth stocks trading at attractive valuations, visit TipRanks Best Stocks to Buy, a newly launched tool that unites all of TipRanks equity insights. Disclaimer: The opinions expressed in this article are solely those of the featured analysts. The content is intended to be used for informational purposes only. It is very important to do your own analysis before making any investment.

Originally posted here:
CRISPR Therapeutics to Participate in the Guggenheim Healthcare Talks 2021 Oncology Day - Yahoo Finance

The study on the global CRISPR and CAS Gene market defines all of the segments together with the market sizing, year-over-year evaluation, and shape and size of the enterprise. Along with those product applications, it also examined whether it reaches up to the end-users or not. This report on this CRISPR and CAS Gene market has given an overall view of the recent technologies used and technological improvements. It also focuses on recent industry trends and which products are quite demanding from a customers perspective.

This report is representing a whole market scenario on a global basis. In this report, we can also find the analysis growth of industries. Through this report, we can easily interpreter the level of market competition, different pricing models, the latest market trends, customer demand, etc. This report acknowledges the revenue model and market expansion of this CRISPR and CAS Gene market. If you want to get that full market information, then this report can help you. It also gives a comprehensive knowledge about the demand and supply graph. Suppose that demand curves moved downward, then from this report, you can know about those factors responsible for its decline.

>>>Get a Sample Copy of the Report at https://www.coherentmarketinsights.com/insight/request-sample/2598

CRISPR and CAS Gene market competitive landscape provides details and data information by players. The report offers comprehensive analysis and accurate statistics on revenue. It also offers detailed analysis supported by reliable statistics on revenue (global and regional level). Details included are company description, major business, company total revenue and the sales, revenue generated in CRISPR and CAS Gene business, the date to enter into the CRISPR and CAS Gene market, CRISPR and CAS Gene product introduction, recent developments, etc.

Some of the key players/Manufacturers involved in the CRISPR and CAS Gene MarketCaribou Biosciences Inc., CRISPR Therapeutics, Mirus Bio LLC, Editas Medicine, Takara Bio Inc., Synthego, Thermo Fisher Scientific, Inc., GenScript, Addgene, Merck KGaA (Sigma-Aldrich), Integrated DNA Technologies, Inc., Transposagen Biopharmaceuticals, Inc., OriGene Technologies, Inc., New England Biolabs, Dharmacon, Cellecta, Inc., Agilent Technologies, and Applied StemCell, Inc.

Detailed Segmentation:

By Product Type:

By Application:

By End User:

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If opting for the Global version of CRISPR and CAS Gene Market analysis is provided for major regions as follows:

North America (The US, Canada, and Mexico)

Europe (the UK, Germany, France, and Rest of Europe)

Asia Pacific (China, India, and Rest of Asia Pacific)

Latin America (Brazil and Rest of Latin America)

Middle East & Africa (Saudi Arabia, the UAE, South Africa, and Rest of Middle East & Africa)

Research and Methodology

For the research, the CRISPR and CAS Gene markets research teams are adopted various high-end techniques. Industry best analysts are worked on this report. They collected data from various reliable sources and have taken samples of different market segments. They utilize both qualitative and quantitative data in this report. All data are based on primary sources, which are focused on the assessment year 2020-2027. For wise decision-making, they have also done SWOT analysis, which can also help them know their predicted future results. This report also helps to develop CRISPR and CAS Gene market growth by improvising its strategic models.

Key Benefits:

This study gives a detailed analysis of drivers and factors limiting the market expansion of CRISPR and CAS Gene

The micro-level analysis is conducted based on its product types, end-user applications, and geographies

Porters five forces model gives an in-depth analysis of buyers and suppliers, threats of new entrants & substitutes and competition amongst the key market players

By understanding the value chain analysis, the stakeholders can get a clear and detailed picture of this CRISPR and CAS Gene market

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Table of Contents

Report Overview: It includes the CRISPR and CAS Gene market study scope, players covered, key market segments, market analysis by application, market analysis by type, and other chapters that give an overview of the research study.

Executive Summary: This section of the report gives information about CRISPR and CAS Gene market trends and shares, market size analysis by region and analysis of global market size. Under market size analysis by region, analysis of market share and growth rate by region is provided.

Profiles of International Players: Here, key players of the CRISPR and CAS Gene market are studied on the basis of gross margin, price, revenue, corporate sales, and production. This section gives a business overview of the players and shares their important company details.

Regional Study: All of the regions and countries analyzed in the CRISPR and CAS Gene market report is studied on the basis of market size by application, the market size by product, key players, and market forecast.

Actual Numbers & In-Depth Analysis, Business opportunities, Market Size Estimation Available in Full Report.

Contacts US:

Mr. ShahCoherent Market Insights,1001 4th Ave,#3200 Seattle, WA 98154, U.S.Phone: US +1-206-701-6702/UK +44-020 8133 4027Email:sales@coherentmarketinsights.com

Original post:
CRISPR and CAS Gene Market Competitive Insights With Global Outlook 2020-2027 | Caribou Biosciences Inc., CRISPR Therapeutics, Mirus Bio LLC, Editas...

New York, Feb. 01, 2021 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Global CRISPR Gene Editing Market: Focus on Products, Applications, End Users, Country Data (16 Countries), and Competitive Landscape - Analysis and Forecast, 2020-2030" - https://www.reportlinker.com/p06018975/?utm_source=GNW Application Agricultural, Biomedical (Gene Therapy, Drug Discovery, And Diagnostics), Industrial, and Other Applications [Genetically Modified Foods (GM Foods), Biofuel, And Animal (Livestock) Breeding] End-User - Academic Institutes and Research Centers, Biotechnology Companies, Contract Research Organizations (CROs), and Pharmaceutical and Biopharmaceutical Companies

Regional Segmentation

North America U.S., Canada Europe Germany, France, Italy, U.K., Spain, Switzerland, and Rest-of-Europe Asia-Pacific China, Japan, India, South Korea, Singapore, Australia, and Rest-of-Asia-Pacific (RoAPAC) Latin America Brazil, Mexico, and Rest-of-the-Latin America Rest-of-the-World

Growth Drivers

Prevalence of Genetic Disorders and Use of Genome Editing Government and Private Funding Technology Advancement in CRISPR Gene Editing

Market Restraints

CRISPR Gene Editing: Off Target Effects and Delivery Ethical Concerns and Implications with Respect to Human Genome Editing

Market Opportunities

Expanding Gene and Cell Therapy Area CRISPR Gene Editing Scope in Agriculture

Key Companies ProfiledAbcam, Inc., Applied StemCell, Inc., Agilent Technologies, Inc., Cellecta, Inc., CRISPR Therapeutics AG, Thermo Fisher Scientific, Inc., GeneCopoeia, Inc., GeneScript Biotech Corporation, Horizon Discovery Group PLC, Integrated DNA Technologies, Inc., Merck KGaA, New England Biolabs, Inc., Origene Technologies, Inc., Rockland Immunochemicals, Inc., Synthego Corporation, System Biosciences LLC, ToolGen, Inc., Takara Bio

Key Questions Answered in this Report: What is CRISPR gene editing? What is the timeline for the development of CRISPR technology? How did the CRISPR gene editing market evolve, and what is its scope in the future? What are the major market drivers, restraints, and opportunities in the global CRISPR gene editing market? What are the key developmental strategies that are being implemented by the key players to sustain this market? What is the patent landscape of this market? What will be the impact of patent expiry on this market? What is the impact of COVID-19 on this market? What are the guidelines implemented by different government bodies to regulate the approval of CRISPR products/therapies? How is CRISPR gene editing being utilized for the development of therapeutics? How will the investments by public and private companies and government organizations affect the global CRISPR gene editing market? What was the market size of the leading segments and sub-segments of the global CRISPR gene editing market in 2019? How will the industry evolve during the forecast period 2020-2030? What will be the growth rate of the CRISPR gene editing market during the forecast period? How will each of the segments of the global CRISPR gene editing market grow during the forecast period, and what will be the revenue generated by each of the segments by the end of 2030? Which product segment and application segment are expected to register the highest CAGR for the global CRISPR gene editing market? What are the major benefits of the implementation of CRISPR gene editing in different field of applications including biomedical research, agricultural research, industrial research, gene therapy, drug discovery, and diagnostics? What is the market size of the CRISPR gene editing market in different countries of the world? Which geographical region is expected to contribute to the highest sales of CRISPR gene editing market? What are the reimbursement scenario and regulatory structure for the CRISPR gene editing market in different regions? What are the key strategies incorporated by the players of global CRISPR gene editing market to sustain the competition and retain their supremacy?

Market OverviewThe development of genome engineering with potential applications proved to reflect a remarkable impact on the future of the healthcare and life science industry.The high efficiency of the CRISPR-Cas9 system has been demonstrated in various studies for genome editing, which resulted in significant investments within the field of genome engineering.

However, there are several limitations, which need consideration before clinical applications.Further, many researchers are working on the limitations of CRISPR gene editing technology for better results.

The potential of CRISPR gene editing to alter the human genome and modify the disease conditions is incredible but exists with ethical and social concerns. The global CRISPR gene editing market was valued at $846.2 million in 2019 and is expected to reach $10,825.1 million by 2030, registering a CAGR of 26.86% during the forecast.

The growth is attributed to the increasing demand in the food industry for better products with improved quality and nutrient enrichment and the pharmaceutical industry for targeted treatment for various diseases. Further, the continued significant investments by healthcare companies to meet the industry demand and growing prominence for the gene therapy procedures with less turnaround time are the prominent factors propelling the growth of the global CRISPR gene editing market.

Research organizations, pharmaceutical and biotechnology industries, and institutes are looking for more efficient genome editing technologies to increase the specificity and cost-effectiveness, also to reduce turnaround time and human errors.Further, the evolution of genome editing technologies has enabled wide range of applications in various fields, such as industrial biotech and agricultural research.

These advanced methods are simple, super-efficient, cost-effective, provide multiplexing, and high throughput capabilities. The increase in the geriatric population and increasing number of cancer cases, and genetic disorders across the globe are expected to translate into significantly higher demand for CRISPR gene editing market.

Furthermore, the companies are investing huge amounts in the research and development of CRISPR gene editing products, and gene therapies. The clinical trial landscape of various genetic and chronic diseases has been on the rise in recent years, and this will fuel the CRISPR gene editing market in the future.

Within the research report, the market is segmented based on product type, application, end-user, and region. Each of these segments covers the snapshot of the market over the projected years, the inclination of the market revenue, underlying patterns, and trends by using analytics on the primary and secondary data obtained.

Competitive LandscapeThe exponential rise in the application of precision medicine on a global level has created a buzz among companies to invest in the development of novel CRISPR gene editing. Due to the diverse product portfolio and intense market penetration, Merck KGaA, and Thermo Fisher Scientific Inc. have been the pioneers in this field and have been the major competitors in this market. The other major contributors of the market include companies such as Integrated DNA Technologies (IDT), Genscript Biotech Corporation, Takara Bio Inc, Agilent Technologies, Inc., and New England Biolabs, Inc.

Based on region, North America holds the largest share of CRISPR gene editing market due to substantial investments made by biotechnology and pharmaceutical companies, improved healthcare infrastructure, rise in per capita income, early availability of approved therapies, and availability of state-of-the-art research laboratories and institutions in the region. Apart from this, Asia-Pacific region is anticipated to grow at the fastest CAGR during the forecast period.

Countries Covered North America U.S. Canada Europe Germany Italy France Spain U.K. Switzerland Rest-of-Europe Asia-Pacific China India Australia South Korea Singapore Japan Rest-of-Asia-Pacific Latin America Brazil Mexico Rest-of-Latin America Rest-of-the-WordRead the full report: https://www.reportlinker.com/p06018975/?utm_source=GNW

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Global CRISPR Gene Editing Market: Focus on Products, Applications, End Users, Country Data (16 Countries), and Competitive Landscape - Analysis and...

Gene Editing Tools Marketresearch report is the new statistical data source added byA2Z Market Research.

Gene Editing Tools Marketresearch is an intelligence report with meticulous efforts undertaken to study the right and valuable information. The data which has been looked upon is done considering both, the existing top players and the upcoming competitors. Business strategies of the key players and the new entering market industries are studied in detail. Well explained SWOT analysis, revenue share and contact information are shared in this report analysis. It also provides market information in terms of development and its capacities.

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Some of the Top companies Influencing in this Market includes:

Thermofisher Scientific, CRISPR Therapeutics, Editas Medicine, NHGRI, Intellia Therapeutics, Merck KGaA.

Various factors are responsible for the markets growth trajectory, which are studied at length in the report. In addition, the report lists down the restraints that are posing threat to the global Gene Editing Tools market. It also gauges the bargaining power of suppliers and buyers, threat from new entrants and product substitute, and the degree of competition prevailing in the market. The influence of the latest government guidelines is also analyzed in detail in the report. It studies the Gene Editing Tools markets trajectory between forecast periods.

Global Gene Editing Tools Market research report offers:

Key Factors Impacting Market Growth:

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Regions Covered in the Global Gene Editing Tools Market Report 2021:The Middle East and Africa(GCC Countries and Egypt)North America(the United States, Mexico, and Canada)South America(Brazil etc.)Europe(Turkey, Germany, Russia UK, Italy, France, etc.)Asia-Pacific(Vietnam, China, Malaysia, Japan, Philippines, Korea, Thailand, India, Indonesia, and Australia)

The cost analysis of the Global Gene Editing Tools Market has been performed while keeping in view manufacturing expenses, labor cost, and raw materials and their market concentration rate, suppliers, and price trend. Other factors such as Supply chain, downstream buyers, and sourcing strategy have been assessed to provide a complete and in-depth view of the market. Buyers of the report will also be exposed to a study on market positioning with factors such as target client, brand strategy, and price strategy taken into consideration.

Key questions answered in the report include:

Table of Content (TOC)

Global Gene Editing Tools Market Report 2021 Growth, Trend and Forecast to 2027

Chapter 1 Gene Editing Tools Market Overview

Chapter 2 Global Economic Impact on Gene Editing Tools Industry

Chapter 3 Global Gene Editing Tools Market Competition by Manufacturers

Chapter 4 Global Production, Revenue (Value) by Region (2014-2020)

Chapter 5 Global Supply (Production), Consumption, Export, Import by Regions (2014-2020)

Chapter 6 Global Production, Revenue (Value), Price Trend by Type

Chapter 7 Global Market Analysis by Application

Chapter 8 Manufacturing Cost Analysis

Chapter 9 Industrial Chain, Sourcing Strategy and Downstream Buyers

Chapter 10 Marketing Strategy Analysis, Distributors/Traders

Chapter 11 Market Effect Factors Analysis

Chapter 12 Global Gene Editing Tools Market Forecast (2021-2027)

Chapter 13 Appendix

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Massive Growth of Gene Editing Tools Market 2021 | Size, Demand, Opportunities & Forecast To 2027 | Thermofisher Scientific, CRISPR Therapeutics,...

CRISPR-wielder Mammoth Biosciences will team up with Agilent Technologies to help launch its upcoming COVID-19 diagnostic test, designed to handle more than 4,000 samples per day.

Applying the gene editing technology allows the system to operate much faster than standard PCR-based molecular lab tests, according to Mammoth. The companys CRISPR-based DETECTR assay uses Cas12 enzymes to identify and tag the coronaviruss specific genomic sequences and provides a visual result that can be read by a machine.

The two companies hope to increase the tests throughput speed even more by connecting it with Agilents automated liquid handling systems and sample readers. Dubbed DETECTR BOOST, the platform aims to process about 1,500 samples over an eight-hour shift.

Blazing a Trail to Clinical Trial Diversity: Four-Part Webinar Series from Syneos Health, Featuring Pharma, Clinical Research and Community Health Leaders

This series will identify obstacles that stifle appropriate patient diversity in trials; unpack the organizational overhaul needed; share how sponsors, patients & investigators have come together to overcome hurdles; and explore how policy innovations can move the industry forward.

"A highly-automated workstation for SARS-CoV-2 testing provides the capacity needed to bring routine, robust testing to the broader market, said David Edwards, associate marketing vice president for Agilents mass spectrometry division. By partnering with Mammoth Biosciences, we will be able to provide a simplified workflow that addresses the specific needs of high-throughput clinical testing laboratories.

Similar methods have been explored for COVID-19 tests that can be read with a smartphone camera and a darkened box using fluorescent molecules that produce a faint glow when matched up with the viruss specific genetic material. Prior to the pandemic, the technology was being developed for HIV testing in low-resource areas.

GlaxoSmithKline has also tapped Mammoth to develop a CRISPR-based coronavirus test that could be available over the counter.

More recently, Mammoth announced a contract with MRIGlobal through the U.S. Defense Advanced Research Projects Agency, known as DARPA, to develop CRISPR-based diagnostics and biosurveillance technologies for the Department of Defense. This includes the development of a hand-held device capable of screening for 10 pathogens simultaneously and a lab system that can spot more than 1,000 targets at once.

RELATED: Pairing CRISPR with a smartphone camera, this COVID-19 test finds results in 30 minutes

Co-founded by CRISPR pioneer and Nobel laureate Jennifer Doudna, Ph.D., Mammoth previously received funding support from the National Institutes of Health through its Shark Tank-esque diagnostics competition known as the Rapid Acceleration of Diagnostics initiative, or RADx. The company said it plans to submit its test for an FDA emergency authorization in the near future.

Mammoths mission is to address challenges across healthcare by harnessing the full potential of the CRISPR platform to read and write the code of life," said Mammoths co-founder and CEO, Trevor Martin, Ph.D. This partnership will help address the need for more widespread testing options for COVID-19, helping to fill the gap in the market as testing labs run into supply issues or reach capacity.

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Mammoth Biosciences teams with Agilent to deliver CRISPR-based coronavirus tests - FierceBiotech

In recent months, even as our attention has been focused on the coronavirus outbreak, there have been a slew of scientific breakthroughs in treating diseases that cause blindness.

Researchers at U.S.-based Editas Medicine and Ireland-based Allergan have administeredCRISPR for the first time to a person with a genetic disease. This landmark treatment uses the CRISPR approach to a specific mutation in a gene linked to childhood blindness. The mutation affects the functioning of the light-sensing compartment of the eye, called the retina, and leads to loss of the light-sensing cells.

According to the World Health Organization,at least 2.2 billion peoplein the world have some form of visual impairment. In the United States, approximately200,000 people suffer from inherited forms of retinal diseasefor which there is no cure. But things have started to change for good. We can now see light at the end of the tunnel.

I am an ophthalmology and visual sciences researcher, and am particularly interested in these advances becausemy laboratory is focusingon designing new and improved gene therapy approaches to treat inherited forms of blindness.

Gene therapy involves inserting the correct copy of a gene into cells that have a mistake in the genetic sequence of that gene, recovering the normal function of the protein in the cell. The eye is an ideal organ for testing new therapeutic approaches, including CRISPR. That is because the eye is the most exposed part of our brain and thus is easily accessible.

The second reason is that retinal tissue in the eye is shielded from the bodys defense mechanism, which would otherwise consider the injected material used in gene therapy as foreign and mount a defensive attack response. Such a response would destroy the benefits associated with the treatment.

In recent years, breakthrough gene therapy studies paved the way to thefirst ever Food and Drug Administration-approved gene therapy drug, Luxturna TM, for a devastating childhood blindness disease,Leber congenital amaurosisType 2.

This form of Leber congenital amaurosis is caused by mutations in a gene that codes for a protein called RPE65. The protein participates in chemical reactions that are needed to detect light. The mutations lessen or eliminate the function of RPE65, which leads to our inability to detect light blindness.

The treatment method developed simultaneously by groups at University of Pennsylvania and at University College London and Moorefields Eye Hospital involvedinserting a healthy copy of the mutated genedirectly into the space between the retina and the retinal pigmented epithelium, the tissue located behind the retina where the chemical reactions takes place. This gene helped the retinal pigmented epithelium cell produce the missing protein that is dysfunctional in patients.

Although the treated eyes showed vision improvement, as measured by the patients ability to navigate an obstacle course at differing light levels,it is not a permanent fix. This is due to the lack of technologies that can fix the mutated genetic code in the DNA of the cells of the patient.

Lately, scientists have been developing a powerful new tool that is shifting biology and genetic engineering into the next phase. This breakthroughgeneeditingtechnology, which is called CRISPR, enables researchers to directly edit the genetic code of cells in the eye and correct the mutation causing the disease.

Children suffering from the disease Leber congenital amaurosis Type 10 endure progressive vision loss beginning as early as one year old. This specific form of Leber congenital amaurosis is caused by a change to the DNA that affects the ability of the gene called CEP290 to make the complete protein. The loss of the CEP290 protein affects the survival and function of our light-sensing cells, called photoreceptors.

One treatment strategy is to deliver the full form of the CEP290 gene using a virus as the delivery vehicle. But the CEP290 gene is too big to be cargo for viruses. So another approach was needed. One strategy was to fix the mutation by using CRISPR.

The scientists at Editas Medicine first showed safety and proof of the concept of the CRISPR strategy in cells extracted from patient skin biopsy and in nonhuman primate animals.

These studies led to the formulation of thefirst ever in human CRISPR gene therapeutic clinical trial. This Phase 1 and Phase 2 trial will eventually assess the safety and efficacy of the CRISPR therapy in 18 Leber congenital amaurosis Type 10 patients. The patients receive a dose of the therapy while under anesthesia when the retina surgeon uses a scope, needle and syringe to inject the CRISPR enzyme and nucleic acids into the back of the eye near the photoreceptors.

To make sure that the experiment is working and safe for the patients, the clinical trial has recruited people with late-stage disease and no hope of recovering their vision. The doctors are also injecting the CRISPR editing tools into only one eye.

An ongoing project in my laboratory focuses on designing a gene therapy approach for the same gene CEP290. Contrary to the CRISPR approach, which can target only a specific mutation at one time, my team is developing an approach that would work for all CEP290 mutations in Leber congenital amaurosis Type 10.

This approach involves usingshorter yet functional forms of the CEP290 proteinthat can be delivered to the photoreceptors using the viruses approved for clinical use.

Gene therapy that involves CRISPR promises a permanent fix and a significantly reduced recovery period. A downside of the CRISPR approach is the possibility of an off-target effect in which another region of the cells DNA is edited, which could cause undesirable side effects, such as cancer. However, new and improved strategies have made such likelihood very low.

Although the CRISPR study is for a specific mutation in CEP290, I believe the use of CRISPR technology in the body to be exciting and a giant leap. I know this treatment is in an early phase, but it shows clear promise. In my mind, as well as the minds of many other scientists, CRISPR-mediated therapeutic innovation absolutely holds immense promise.

In another study just reported in the journal Science, German and Swiss scientists have developeda revolutionary technology, which enables mice and human retinas to detect infrared radiation. This ability could be useful for patients suffering from loss of photoreceptors and sight.

The researchers demonstrated this approach, inspired by the ability of snakes and bats to see heat, by endowing mice and postmortem human retinas with a protein that becomes active in response to heat. Infrared light is light emitted by warm objects that is beyond the visible spectrum.

The heat warms a specially engineered gold particle that the researchers introduced into the retina. This particle binds to the protein and helps it convert the heat signal into electrical signals that are then sent to the brain.

In the future, more research is needed to tweak the ability of the infrared sensitive proteins to different wave lengths of light that will also enhance the remaining vision.

This approach is still being tested in animals and in retinal tissue in the lab. But all approaches suggest that it might be possible to either restore, enhance or provide patients with forms of vision used by other species.

Hemant Khanna is an Associate Professor of Ophthalmology at the University of Massachusetts Medical School. His lab investigates molecular and cell biological bases of severe photoreceptor degenerative disorders, such as Retinitis Pigmentosa (RP) and Leber Congenital Amaurosis (LCA). Find Hemant on Twitter @khannacilialab

A version of this article was originally published at the Conversation and has been republished here with permission. The Conversation can be found on Twitter @ConversationUS

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Inherited blindness has a new cure, thanks to CRISPR - Genetic Literacy Project

The mosquitoes that carry zika, like Aedes aegypti, are considered by all but the most activist ecologists to be useless disease vectors. There is nothing they do in nature that isn't easily done by other mosquitoes and they can safely join the 99.999999999% of species that have gone extinct without causing a cascade of doom.

Seriously, Send me your hate mail, @ me on Twitter, try to cancel me, I don't care, that is absolutely correct. They are ecologically useless and have survived despite that, because evolution is not always fair.

After years of study across two different administrations, the EPA signed off on a plan that would allow release of genetically engineered mosquitoes that can't reproduce - since their range is limited their area of effect would be only what we want, like the Florida Keys. In the Los Angeles Times years earlier, I had debunked the claims of an anti-science Florida "Karen" who was convinced biologists didn't know what they were doing and we were all going to be dead, the environmental equivalent of that lawyer who filed a lawsuit claiming the Large Hadron Collider would create a black hole and swallow the planet. Lawyer-run groups that wrap themselves in the flag of environmentalism have also been raising money opposing natural control of mosquitoes recently, conspiratorially insisting Trump controlled every career scientist in government so it can't be science if an approval was made during his four years. They argue using the blanket denier-for-hire mantra; there could be unintended consequences.

Like what? The outcomes they invent are more magic than mosquito reality, such as that Frankenflies will escape and create mutant offspring. In science realty, male Aedes aegypti mosquitoes are harmless, they don't bite us. These time-limited males mate with females and simply can't have pestilence offspring.

A new study thinks it can fill the gap in the concern activists say they have.(1) What the scientists behind the paper use as their selling point is they won't have the "environmental complications" of GMO mosquitoes that can't reproduce, because by using CRISPR they have created mosquitoes that can't replicate the Zika virus at all, so females won't send it to humans when they bite.

Scientifically, that is great, but people with experience fighting the war on science know that it won't change anything. Science is a veneer for environmentalists. Activists hate CRISPR as much as they hate GMOs or RNAi or everything except the organic food created using Mutagenesis chemical and radiation baths. They will oppose this because the trait that keeps them from transmitting zika is heritable. It can be passed down. That will be spun into world-class "environmental complications" and Frankenbug unintended consequences so dumb even that terrible Dustin Hoffman movie Outbreak didn't try to use them.

If this gets close to testing, and the propaganda campaign against it ensues, the authors will then scramble to assure the public that Aedes aegypti proteins are not expressed salivary glands so humans are not at risk, this cannot mutate, etc., but they are not equipped to combat the disinformation and misinformation of a $2 billion-a-year marketing machine. By then half the public is already against it, the same way Democrats oppose the Keystone Pipeline despite Obama administration scientists clearing it - twice, the second time after Obama staffers made them do it again because they wanted to ban it.

That's a problem down the road, though. For now, applied use isn't even a discussion, it is just intriguing basic research on how we might solve an infectious disease problem in a way that might make people feel better than pesticides.

The problem is that environmentalists have turned all of biology into their new "Silent Spring" fundraisers. That's ironic, because Rachel Carson believed biological solutions like this were the ideal replacement for pesticides.

NOTES:

(1) Which falls into the real trap of activists; it is not about science, they claim you can fix the thing they object to but they will then move onto another. See Golden Rice. Academics believed that the concern was really about corporate control over food, and was not a war on science itself, so they created a public domain fortified bowl of rice. They quickly learned it was not about Monsanto at all. Having no corporate sponsor, they had no lawyers, but environmentalists are lawyer-heavy, and blocked its approval easily, while their marketing groups and academic allies insisted it would not work anyway.

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An Alternate Approach To Stopping Mosquitoes That Spread Zika - Using CRISPR To Make Them Resistant To Carrying It - Science 2.0

Global CRISPR and CAS GeneMarket, By Product Type (Vector-based Cas and DNA-free Cas), By Application (Genome Engineering, Disease models, Functional Genomics, Knockdown/activation, and Other Applications), By End User (Biotechnology and Pharmaceutical Companies,Academic Government Research Institutes, and Contract Research Organizations), and By Region (North America, Latin America, Europe, Asia Pacific, Middle East, and Africa) was valued at US$ 1,388.1 million in 2017, and is projected to exhibit a CAGR of 20.8% over the forecast period (2018 2026).

Manufacturers in the CRISPR and CAS gene are collaborating with many companies for sponsoring clinical trials. Editas Medicine has licensed CRISPR and other gene editing patent rights from the Broad Institute, the Massachusetts Institute of Technology (MIT), Harvard University, and others. In March 2017, Editas reportedly entered into an agreement with Irish pharmaceutical company Allergan under, which Editas was to receive a US$ 90 million up-front payment for an option to license up to five preclinical programs targeting eye disease. Moreover, various organizations are also focusing on new clinical trials for the CRISPR and CAS gene for cancer treatment. In 2018, CRISPR Therapeutics and Vertex launched the first in-human clinical trial of CRISPR genome editing technology sponsored by U.S. companies. The trial is testing an experimental therapy for the blood disorder -thalassemia in Regensburg, Germany.

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Increasing research and studies regarding the CRISPR and CAS gene technology is majorly driving the growth of CRISPR and CAS gene market. In 2017, Editas partnered with Juno Therapeutics for cancer-related research using CRISPR. Under the terms of the agreement, Juno had to pay Editas an initial payment of US$ 25 million, in which up to US$ 22 million will be used in research support for three programs over five years. Editas has also engaged in a three-year research and development (R&D) collaboration deal with San Raffaele Telethon Institute for Gene Therapy to research and develop next generation stem cell and T-cell therapies for the treatment of rare diseases.

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CRISPR and CAS Gene Market to Score Past US$ 7603.8 Million Valuation by 2027: CMI KSU | The Sentinel Newspaper - KSU | The Sentinel Newspaper

Like many other advances in science and medicine, CRISPR was inspired by nature. In this case, the idea was borrowed from a simple defense mechanism found in some microbes, such as bacteria.

To protect themselves against invaders like viruses, these microbes capture snippets of the intruders DNA and store them away as segments called CRISPRs, or clustered regularly interspersed short palindromic repeats. If the same germ tries to attack again, those DNA segments (turned into short pieces of RNA) help an enzyme called Cas find and slice up the invaders DNA.

After this defense system was discovered, scientists realized that it had the makings of a versatile gene-editing tool. Within a handful of years, multiple groups had successfully adapted the system to edit virtually any section of DNA, first in the cells of other microbes, and then eventually in human cells.

There are still a lot of questions about all the ways that CRISPR might beput to use in cancer researchand treatment. But one thing is for certain: The field is moving incredibly fast and new applications of the technology are constantly popping up.

People are still improving CRISPR methods, Dr. [Jerry] Li said. Its quite an active area of research and development. Im sure that CRISPR will have even broader applications in the future.

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Diseases once thought incurable are now on the cusp of treatments. It's because of CRISPR. Here's a primer - Genetic Literacy Project

Through the social and economic disruption that COVID-19 caused in 2020, the biomedical research community rose to the challenge and accomplished unprecedented feats of scientific acumen. With a new year ahead of us, even as the pandemic grinds on, we at The Scientist thought it was an opportune time to ask what might be on the life science innovation radar for 2021 and beyond. We tapped three members of the independent judging panel that helped name our Top 10 Innovations of 2020 to share their thoughts (via email) on the year ahead.

Paul Blainey: Value is shifting from the impact of individual technologies (mass spectrometry, cloning, sequencing, PCR, induced pluripotent stem cells, next generation sequencing, genome editing, etc.) to impact across technologies. In 2021, I think researchers will increasingly leverage multiple technologies together in order to generate new insights, as well as become more technology-agnostic as multiple technologies present plausible paths toward research goals.

Kim Kamdar: Partially in reaction to the COVID-19 pandemic, one 2021 headline will be the continued innovation focused on consumerization of healthcare, which is redefining how consumers engage with providers across each stage of care. Consumers are even selective about their healthcare choices now, and the retail powerhouses like CVS and Walmart have and will continue to develop solutions to meet the needs of their customers. While this was already underway prior to the pandemic, the crisis has spurred on this activity with the goal of making healthcare more accessible and affordable and ultimately delivering on better health outcomes for all Americans.

Robert Meagher: I think this is easymRNA delivery. This is something that has been in development for years for numerous applications, but the successful development and FDA emergency use authorization of two COVID-19 vaccines based on this technology shines a very bright spotlight on this technology. The vaccine trials and now widespread use of the vaccines will give developers a lot of data about the technology, and sets a baseline for understanding safety and side effects when considering future therapeutic applications outside of infectious disease.

PB:Single-cell technology is here to stay, although its use will continue to change. One analogy to be drawn is the shift we saw from the popularity ofde novo genome sequencing (during the human genome project and the early part of the NGS [next-generation sequencing] era to the rich array of re-sequencing applications practiced today. I expect new ways to use single-cell technology will continue to be discovered for some time to come.

KK: Innovation in single-cell technology has the potential to transform biological research driving to a level of resolution that provides a more nuanced picture of complex biology. Cost has been a key barrier for broader adoption of single-cell analysis. As better technology is developed, cost will be reduced and there will be an explosion in single-cell research. This dynamic will also allow for broader adoption of single-cell technology from translational research to clinical applications particularly in oncology and immunology.

RM: Yesthere is continuing innovation in this space, and room for continued innovation. One area that we have seen development recently, and I see it continuing, is to study single cells not just in isolation, but coupled with spatial information: understanding single cells and their interactions with their neighbors. I also wonder if the COVID-19 pandemic will spur increased interest in applying single-cell techniques to problems in infectious disease, immunology, and microbiology. A lot of the existing methods for single-cell RNA analysis (for example) work well for human or mammalian cells, but dont work for bacteria or viruses.

PB: The promises of CRISPR and gene editing are extraordinary. I cant wait to see how that field continues to develop.

KK: Much of the CRISPR technology focus since it was unveiled in 2012 has been on its utility to modify genes in human cells with the goal of treating genetic disease. More recently, scientists have shown the potential of using the CRISPR gene-editing technology for treatment of viral disease (essentially a programmable anti-viral that could be used to treat diseases like HIV, HBV, SARS, etc. . . .). These findings, published in Nature Communications, showed that CRISPR can be used to eliminate simian immunodeficiency virus (SIV) in rhesus macaque monkeys. If replicated in humans, in studies that will be initiated this year, CRISPR could be utilized to address HIV/AIDS and potentially make a major impact by moving a chronic disease to one with a functional cure.

PB: New therapeutic modalities that expand the addressable set of diseases are particularly exciting. Cell-based therapies offer versatile platforms for biological engineering that leverage the power of human biology. It is also encouraging to see somatic cell genome editing technology advance toward the clinic for the treatment of serious diseases.

The level of innovation that occurred in 2020 to combat COVID-19 will provide a more rapid, focused, and actionable reaction to future pandemics.

Kim Kamdar, Domain Associates

RM: Besides the great success with mRNA-based vaccines that sets the stage for other clinical technologies based on mRNA delivery, the other area that is really in the spotlight this year is diagnostics. There are a lot of labs and companies, both small and large, that have some really innovative products and ideas for portable and point-of-care diagnostics. For a long time, this was often thought of in terms of a problem for the developing world, or resource-limited locations: think, for example, of diagnostics for neglected tropical diseases. But the COVID-19 pandemic and the associated need for diagnostic testing on a massive scale has caused us to rethink what resource-limited means, and to understand the challenge posed by bottlenecks in supply chains, skilled personnel, and high-complexity laboratory facility. There has been a lot of foundational research over the past couple of decades in rapid, portable, easy-to-use diagnostics, but translating these to clinically useful products often seemed to stall, I suspect for lack of a lucrative market for such tests. But we are now starting to see FDA [emergency use authorization for] home-based tests and other novel diagnostic technologies to address needs with the COVID-19 pandemic, and I suspect that this paves the way for these technologies to start being applied to other diagnostic testing needs.

PB: Seeing the suffering and destruction wrought by COVID-19, it is obvious that we need to be prepared with more extensive, equitable, and better-coordinated response plans going forward. While rapid vaccine development and testing were two bright spots last year, there are so many important areas that demand progress. As we learn about how important details become in a crisisno matter how small or mundanediagnostic technologies and the calibration of public health measures are two areas that merit major focus.

KK: The life science community response to the COVID-19 pandemic has already proven to be light-years ahead of previous responses particularly in areas such as vaccine development and diagnostics. It took more than a year to sequence the genome of the SARS virus in 2002. The COVID-19 genome was sequenced in under a month from the first case being identified. Scientists and clinicians were able to turn that initial information to multiple approved vaccines at a blazing speed. Utilizing messenger RNA (mRNA) as a new therapeutic modality for vaccine development has now been validated. Vaccine science has been forever changed. The pandemic has also focused a much-needed level of attention to diagnostics, forcing a rethink of how to increase access, affordability, and actionability of diagnostic testing. The level of innovation that occurred in 2020 to combat COVID-19 will provide a more rapid, focused, and actionable reaction to future pandemics. In addition, the elevation of a science advisor (Dr. Eric Lander) to a cabinet level position in the Biden administration bodes well for our future ability to ground in data and as President Biden himself framed, refresh and reinvigorate our national science and technology strategy to set us on a strong course for the next 75 years, so that our children and grandchildren may inhabit a healthier, safer, more just, peaceful, and prosperous world.

RM: One thing that really kick-started research to address COVID-19 was the early availability of the complete genome sequence of the SARS-CoV-2 virus, and the ongoing timely deposition of new sequences in nearreal-time as isolates were sequenced. This is in contrast to cases where deposition of large number of sequences may lag an outbreak by months or even years. I foresee the nearreal-time sharing of sequence information to become the new standard. Making the virus itself widely and inexpensively available, in inactivated form, as well as well-characterized synthetic viral RNA standards and proteins also helped spur research.

A trend Im less fond of is the rapid publication of nonpeer reviewed results as preprints online. Theres a great benefit to getting new information out to the community ASAP, but unfortunately I think the rush to get preprints up in some cases results in spreading misleading information. This problem is compounded with uncritical, breathless press releases accompanying the posting of preprints, as opposed to waiting for peer-review acceptance of a manuscript to issue a press release. I think the solution may lie in journals considering innovative approaches to speeding up peer review, or a way to at least perform a basic check for rigor prior to posting a preliminary version of the manuscript. Right now the extremes are: post an unreviewed preprint, or wait months or even years with multiple rounds of peer review including extensive additional experiments to satisfy the curiosity of multiple reviewers for high impact publications. Is there a way to prevent manuscripts from being published as preprints with obvious methodological errors or errors in statistical analysis, while also enabling interesting, well-done yet not fully polished manuscripts to be available to the community?

Paul Blaineyis an associate professor of biological engineering at MIT and a core member of the Broad Institute of MIT and Harvard University. The Blainey lab integrates new microfluidic, optical, molecular, and computational tools for application in biology and medicine. The group emphasizes quantitative single-cell and single-molecule approaches, aiming to enable studies that generate data with the power to reveal the workings of natural and engineered biological systems across a range of scales. Blainey has a financial interest in several companies that develop and/or apply life science technologies: 10X Genomics, GALT, Celsius Therapeutics, Next Generation Diagnostics, Cache DNA, and Concerto Biosciences.

Kim Kamdaris managing partner at Domain Associates, a healthcare-focused venture fund creating and investing in biopharma, device, and diagnostic companies. She began her career as a scientist and pursued drug-discovery research at Novartis/Syngenta for nine years.

Robert Meagheris a principal member of Technical Staff at Sandia National Laboratories. His main research interest is the development of novel techniques and devices for nucleic acid analysis, particularly applied to problems in infectious disease, biodefense, and microbial communities. Most recently this has led to approaches for simplified molecular diagnostics for emerging viral pathogens that are suitable for use at the point of need or in the developing world. Meaghers comments represent his professional opinion but do not necessarily represent the views of the US Department of Energy or the United States government.

See the article here:
Experts Predict the Hottest Life Science Tech in 2021 and Beyond - The Scientist

Are you the type of investor who patiently plays the long game, or are you the more impulsive type? While it's easy to give in to the temptation to buy and sell your stocks based on short-term price movements, you'll see the largest gains when you buckle up for the long haul. Five years might seem like a long time to wait for your purchases to pay off, but when it comes to biotech stocks, practiced patience can pay off big.

All of the stocks below have recently met key milestones in their drug development efforts. More importantly, each is planning on making even bigger breakthroughs on the five-year horizon, so they're worth buying and holding until then. Given that these companies are still relatively early-stage and the risks of failure remain high, it'll be prudent for investors to diversify across all five rather than going all-in on any single stock.

Image source: Getty Images.

Late last year, CRISPR Therapeutics (NASDAQ:CRSP) reported favorable results from early-stage clinical trials of its CTX001 gene-editing therapy for beta thalassemia and sickle cell disease. That's great news, as CTX001 is the company's most advanced program. Over the next five years, CTX001 will likely continue to move forward in clinical trials.

The same may be true for four of its immuno-oncology programs that are currently in clinical development. Elsewhere in the pipeline, its regenerative gene therapy for diabetes mellitus will enter its phase 1/2 clinical trials in 2021, and potentially conclude them in the following years. It needs that time to prove that its products are safe and effective.

Lexicon Pharmaceuticals (NASDAQ:LXRX) makes sotagliflozin, which is currently approved to treat type 1 diabetes in the EU. Soon, it will likely conclude its registration with U.S.-based regulators and initiate sales shortly thereafter. The company is also conducting phase 3 clinical trials for the drug to see if it's effective at treating heart failure. If successful, Lexicon will have a steady stream of revenue, and it could reach profitability in the next few years. Its project for diabetic peripheral neuropathic pain in phase 2 clinical trials could also drive shareholder returns even further, assuming it too gets approved.

Fate Therapeutics (NASDAQ:FATE) may not have any products approved for sale, but its collection of immuno-oncology treatments derived from pluripotent stem cells has already made its stock a high performer over the last year. While the stock is doubtlessly a bit expensive at the moment considering how far away the company is from recurring revenue, the company is establishing itself as a leader in the stem cell-derived therapeutics space, which will pay off down the line.

Fate currently has seven projects that are currently in phase 1 clinical trials, which means that it'll likely have at least one of those advancing into the final stages of the process by 2026.

CRSP data by YCharts

Jounce Therapeutics (NASDAQ:JNCE) focuses on developing cancer immunotherapies that address underserved patient populations, and it's been a great stock to own over the last year. It'll report two sets of data from a pair of its clinical trials in the second half of 2021, which could bolster a higher stock price.

Through the next few years, Jounce plans to submit an Investigational New Drug (IND) filing to regulators every 12 to 18 months, paving the way for new early-stage clinical trials. With innovation at such a rapid pace, investors will have plenty of growth catalysts to look forward to, assuming Jounce's projects continue to mature.

Corcept Therapeutics (NASDAQ:CORT) is special because it's already profitable, and it has a larger and more advanced pipeline than the other companies listed here. It makes the drug Korlym, a treatment for Cushing syndrome, and it's also exploring in phase 3 trials another drug for the same indication. The company also has a late-stage pancreatic cancer therapy in development, not to mention a handful of other mid-stage oncology and metabolic disease projects.

If you're a bit worried that the other biotechs on this list might not pay off, you can take heart in that Corcept is considerably safer. Though there's still no guarantee that its pipeline projects will turn into new revenue streams anytime soon, its quarterly revenue is growing by 6% year over year. And it barely has any debt, with a scant $3.03 million owed. In other words, this company's story over the next five years will be about using its proceeds from drug sales to invest heavily in its future opportunities for growth. For a biotech investor, there's no better position to be in.

Read the original here:
Got $5,000 and 5 Years to Wait? Buy These 5 Hot Biotech Stocks Now - Motley Fool

Even after a decade of treating patients with hematologic malignancies with chimeric antigen receptor (CAR) T-cell (CAR-T) therapies, researchers are still trying to understand why most patients eventually relapse. Equally puzzling to some scientists is the question of these cellular interventions cause lasting remission at all in many patients.

Given that typical cancers consist of diverse cells, including those that do not express the antigens targeted by CAR-T cells, one would expect relapses through antigen escape to be much more common than currently observed in practice, explained Joshua Brody, MD, director of the Lymphoma Immunotherapy Program at The Tisch Cancer Institute at Mount Sinai in New York, New York. In CAR-T cells, maybe [only] 40% of patients have this antigen escape problem. Its weird to us that its not 100%, he said.

For instance, even though most patients with B-cell acute lymphocytic leukemia (ALL) possess over 1% of CD19-negative cells at diagnosis, the incidence of relapse after CD19-targeted therapy only approximates 20%.1,2 And, in patients with diffuse large B-cell lymphoma (DLBCL), similar response rates to CAR-T cell therapy have been observed regardless of tumoral expression of CD19.3 To Brody and colleagues, such observations suggest that CAR-T cells use mechanisms independent of antigen targeting to eliminate tumor cells.

A study published by Brodys team and collaborators at Kite Pharma in Cancer Discovery in December 2020 offered one explanation.4 Experiments in animal models suggested that CAR-T cells can kill off-target cells that are in the vicinity of the cells theyre designed to target, offering the first in vivo proof of localized bystander killing. This off-target effect is mediated by the interaction between the protein Fasa cell death receptor expressed on many cellular surfacesand its ligand, which is present on T cells. In fact, tumoral expression of Fas was predictive of survival in patients with DLBCL who were treated with anti-CD19 CAR-T cell therapy in the phase 1/2 ZUMA-1 trial (ClinicalTrials.gov identifier:NCT02348216).

I think its becoming more and more [clear] that the CAR-Ts, in addition to their CAR interaction with the tumor antigen, rely on the Fas-Fas ligandinteraction to exert their killing. The next question is, how do we manipulate these pathways safely to make CARs more potent? said Saad J. Kenderian, MB, ChB, a consultant in the division of hematology in the department of internal medicine at the Mayo Clinic in Rochester, Minnesota, who was not involved in the study.

The new research was the result of a serendipitous observation made during a clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein 9 (Cas9) screen exploring the genes that tumor cells use to either resist or facilitate cytotoxic T cell killing. The team noticed in cell culture that T cells engineered to target a specific protein would not only kill the on-target lymphoma cells expressing that protein, but also the cells that did not.

Further experiments pointed to Fas as a mediator of this process. When the researchers experimentally removed the gene encoding Fas from cultured lymphoma cells, they noticed that this protected both on-target and off-target cells. This was the case even as T-cells emitted the cell death-inducing molecules granzyme and perforin, which are thought to represent the main method of killing.

T cells require an interaction with their target antigen to kill a cell via the perforin and granzyme mechanism, but as long as off-target cells are in the direct vicinity of on-target cells, Brodys results suggest that T-cells can eliminate them via the Fas-dependent mechanism. Essentially, you kill the target cell youre going after and just to be safe, you [also] kill the cell next door, he said.

Further cell culture and mouse experiments bolstered this hypothesis. In one experiment, mice treated with murine CD19 CD3-CD28 CAR-T cells had similar rates of survival irrespective of whether their lymphoma tumors consisted of mixed antigen-positive and negative cells or only of antigen-expressing cells. However, ifwe gave Fas ligand-blocking antibody, then those mice died sooner, Brody added.

See the article here:
Bystander Killing Could Be Key Factor in CAR-T Success in Non-Hodgkin Lymphoma - Cancer Therapy Advisor

Alongside Dennis vanEngelsdorp, associate professor at the University of Maryland (UMD) in Entomology named for the fifth year in a row for his work in honey bee and pollinator health, Yiping Qi, associate professor in Plant Science, represented the College of Agriculture & Natural Resources on the Web of Science 2020 list of Highly Cited Researchers for the first time. Qi is already making waves in 2021 with a new high-profile publication in Nature Plants introducing SpRY, a newly engineered variant of the famed gene editing tool CRISPR-Cas9. SpRY essentially removes the barriers of what can and can't be targeted for gene editing, making it possible for the first time to target nearly any genomic sequence in plants for potential mutation.

"It is an honor, an encouragement, and a recognition of my contribution to the science community," says Qi of his distinction as a 2020 Web of Science Highly Cited Researcher. "But we are not just making contributions to the academic literature. In my lab, we are constantly pushing new tools for improved gene editing out to scientists to make an impact."

With SpRY, Qi is especially excited for the limitless possibilities it opens up for genome editing in plants and crops. "We have largely overcome the major bottleneck in plant genome editing, which is the targeting scope restrictions associated with CRISPR-Cas9. With this new toolbox, we pretty much removed this restriction, and we can target almost anywhere in the plant genome."

The original CRISPR-Cas9 tool that kicked off the gene editing craze was tied to targeting a specific short sequence of DNA known as a PAM sequence. The short sequence is what the CRISPR systems typically use to identify where to make their molecular cuts in DNA. However, the new SpRY variant introduced by Qi can move beyond these traditional PAM sequences in ways that was never possible before.

"This unleashes the full potential of CRISPR-Cas9 genome editing for plant genetics and crop improvement," says an excited Qi. "Researchers will now be able to edit anywhere within their favorable genes, without questioning whether the sites are editable or not. The new tools make genome editing more powerful, more accessible, and more versatile so that many of the editing outcomes which were previously hard to achieve can now be all realized."

According to Qi, this will have a major impact on translational research in the gene editing field, as well as on crop breeding as a whole. "This new CRISPR-Cas9 technology will play an important role in food security, nutrition, and safety. CRISPR tools are already widely used for introducing tailored mutations into crops for enhanced yield, nutrition, biotic and abiotic stress resistance, and more. With this new tool in the toolbox, we can speed up evolution and the agricultural revolution. I expect many plant biologists and breeders will use the toolbox in different crops. The list of potential applications of this new toolbox is endless."

Read the complete research at http://www.sciencedaily.com.

University of Maryland. "Plant genome editing expanded with newly engineered variant of CRISPR-Cas9: New study introduces SpRY to enable the mutation of nearly any genomic sequence in plants." ScienceDaily.

See the article here:
Study on plant genome editing with new variant of CRISPR-Cas9 - hortidaily.com

Phylogenetic trees, starting with an individual cancer cell. Each color represents a different location in the body. A very colorful tree shows a highly metastatic phenotype, where a cells descendants jumped many times between different tissues. A tree that is primarily one color represents a less metastatic cell. Credit: Jeffrey Quinn/Whitehead Institute

Using CRISPR technology, researchers are tracking the lineage of individual cancer cells as they proliferate and metastasize in real-time.

When cancer is confined to one spot in the body, doctors can often treat it with surgery or other therapies. Much of the mortality associated with cancer, however, is due to its tendency to metastasize, sending out seeds of itself that may take root throughout the body. The exact moment of metastasis is fleeting, lost in the millions of divisions that take place in a tumor. These events are typically impossible to monitor in real time, says Jonathan Weissman, MIT professor of biology and Whitehead Institute for Biomedical Research member.

Now, researchers led by Weissman, who is also an investigator with the Howard Hughes Medical Institute, have turned a CRISPR tool into a way to do just that. In a paper published on January 21, 2021, in Science, Weissmans lab, in collaboration with Nir Yosef, a computer scientist at the University of California at Berkeley, and Trever Bivona, a cancer biologist at the University of California at San Francisco, treats cancer cells the way evolutionary biologists might look at species, mapping out an intricately detailed family tree. By examining the branches, they can track the cells lineage to find when a single tumor cell went rogue, spreading its progeny to the rest of the body.

With this method, you can ask questions like, How frequently is this tumor metastasizing? Where did the metastases come from? Where do they go? Weissman says. By being able to follow the history of the tumor in vivo, you reveal differences in the biology of the tumor that were otherwise invisible.

Scientists have tracked the lineages of cancer cells in the past by comparing shared mutations and other variations in their DNA blueprints. These methods, however, depend to a certain extent on there being enough naturally occurring mutations or other markers to accurately show relationships between cells.

Thats where Weissman and co-first authors Jeffrey Quinn, then a postdoc in Weissmans lab, and Matthew Jones, a graduate student in Weissmans lab, saw an opportunity to use CRISPR technology specifically, a method developed by Weissman Lab member Michelle Chan to track embryo development to facilitate tracking.

Instead of simply hoping that a cancer lineage contained enough lineage-specific markers to track, the researchers decided to use Chans method to add in markers themselves. Basically, the idea is to engineer a cell that has a genomic scratchpad of DNA, that then can be written on using CRISPR, Weissman says. This writing in the genome is done in such a way that it becomes heritable, meaning a cells grand-offspring would have the writing of its parent cells and grandparent cells recorded in its genome.

To create these special scratchpad cells, Weissman engineered human cancer cells with added genes: one for the bacterial protein Cas9 the famed molecular scissors used in CRISPR genome editing methods others for glowing proteins for microscopy, and a few sequences that would serve as targets for the CRISPR technology.

They then implanted thousands of the modified human cancer cells into mice, mimicking a lung tumor (a model developed by collaborator Bivona). Mice with human lung tumors often exhibit aggressive metastases, so the researchers reasoned they would provide a good model for tracking cancer progression in real time.

As the cells began to divide, Cas9 made small cuts at these target sites. When the cell repaired the cuts, it patched in or deleted a few random nucleotides, leading to a unique repair sequence called an indel. This cutting and repairing happened randomly in nearly every generation, creating a map of cell divisions that Weissman and the team could then track using special computer models that they created by working with Yosef, a computer scientist.

Tracking cells this way yielded some interesting results. For one thing, individual tumor cells were much different from each other than the researchers expected. The cells the researchers used were from an established human lung cancer cell line called A549. Youd think they would be relatively homogeneous, Weissman says. But in fact, we saw dramatic differences in the propensity of different tumors to metastasize even in the same mouse. Some had a very small number of metastatic events, and others were really rapidly jumping around.

To find out where this heterogeneity was coming from, the team implanted two clones of the same cell in different mice. As the cells proliferated, the researchers found that their descendants metastasized at a remarkably similar rate. This was not the case with the offspring of different cells from the same cell line the original cells had apparently evolved different metastatic potentials as the cell line was maintained over many generations.

The scientists next wondered what genes were responsible for this variability between cancer cells from the same cell line. So they began to look for genes that were expressed differently between nonmetastatic, weakly metastatic, and highly metastatic tumors.

Many genes stood out, some of which were previously known to be associated with metastasis although it was not clear whether they were driving the metastasis or simply a side effect of it. One of them, the gene that codes for the protein Keratin 17, is much more strongly expressed in low metastatic tumors than in highly metastatic tumors. When we knocked down or overexpressed Keratin 17, we showed that this gene was actually controlling the tumors invasiveness, Weissman says.

Being able to identify metastasis-associated genes this way could help researchers answer questions about how tumors evolve and adapt. Its an entirely new way to look at the behavior and evolution of a tumor, Weissman says. We think it can be applied to many different problems in cancer biology.

Weissmans CRISPR method also allowed the researchers to track with more detail where metastasizing cells went in the body, and when. For example, the progeny of one implanted cancer cell underwent metastasis five separate times, spreading each time from the left lung to other tissues such as the right lung and liver. Other cells made a jump to a different area, and then metastasized again from there.

These movements can be mapped neatly in phylogenetic trees (see image), where each color represents a different location in the body. A very colorful tree shows a highly metastatic phenotype, where a cells descendants jumped many times between different tissues. A tree that is primarily one color represents a less metastatic cell.

Mapping tumor progression in this way allowed Weissman and his team to make a few interesting observations about the mechanics of metastasis. For example, some clones seeded in a textbook way, traveling from the left lung, where they started, to distinct areas of the body. Others seeded more erratically, moving first to other tissues before metastasizing again from there.

One such tissue, the mediastinal lymph tissue that sits between the lungs, appears to be a hub of sorts, says co-first author Jeffrey Quinn. It serves as a way station that connects the cancer cells to all of this fertile ground that they can then go and colonize, he says.

Therapeutically, the discovery of metastasis hubs like this could be extremely useful. If you focus cancer therapies on those places, you could then slow down metastasis or prevent it in the first place, Weissman says.

In the future, Weissman hopes to move beyond simply observing the cells and begin to predict their behavior. Its like with Newtonian mechanics if you know the velocity and position and all the forces acting on a ball, you can figure out where the ball is going to go at any time in the future, Weissman says. Were hoping to do the same thing with cells. We want to construct essentially a function of what is driving differentiation of a tumor, and then be able to measure where they are at any given time, and predict where theyre going to be in the future.

The researchers are optimistic that being able to track the family trees of individual cells in real time will prove useful in other settings as well. I think that its going to unlock a whole new dimension to what we think about as a measurable quantity in biology, says co-first author Matthew Jones. Thats whats really cool about this field in general is that were redefining whats invisible and what is visible.

Reference: Single-cell lineages reveal the rates, routes, and drivers of metastasis in cancer xenografts by Jeffrey J. Quinn, Matthew G. Jones, Ross A. Okimoto, Shigeki Nanjo, Michelle M. Chan, Nir Yosef, Trever G. Bivona and Jonathan S. Weissman, 21 January 2021, Science.DOI: 10.1126/science.abc1944

Original post:
Using CRISPR Genetic Technology to Catch Cancer in the Act - SciTechDaily

Two different gene therapies have been used to mitigate a mechanism underlying development of sickle cell disease (SCD) and transfusion-dependent -thalassemia (TDT), and both have demonstrated clinical success in separate, concurrently published trials.

The hemoglobinopathies manifest after fetal hemoglobin synthesis is replaced by adult hemoglobin in individuals who have inherited a mutation in the hemoglobin subunit gene (HBB).Identifying factors in the conversion from fetal to adult hemoglobin synthesis, however, has provided potential targets for therapeutic intervention.

Gene therapy that can safely arrest or reduce the conversion offers the potential for a one-time treatment to obviate the need for lifetime transfusions and iron chelation for patients with TDT, and the pain management, transfusions and hydroxyurea administration for those with SCD.

Two groups of investigators have now reported in The New England Journal of Medicine that, using different gene therapy techniques that target the transcription factor, BCL11a, involved in the globin switching, they have improved clinical outcomes in patients with TDT and with SCD.

In an editorial in the issue featuring the 2 studies, Mark Walters, MD, Blood and Marrow Transplant Program, University of California, San Francisco-Benioff Children's Hospital, welcomed the breakthroughs.

"These trials herald a new generation of broadly applicable curative treatments for hemoglobinopathies," Walters wrote.

In one clinical trial with 2 patients, one with TDT and the other with SCD, Haydar Frangoul, MD, MS, Medical Director, Pediatric Hematology/Oncology, Sarah Cannon Center for Blood Cancer at the Children's Hospital at Tristar Centennial, and colleagues administered CRISPR-Cas9 gene edited hematopoietic stem and progenitor cells (HSPCs) with reduced BCL11A expression in the erythroid lineage.

The product, CTX001, had been shown in preclinical study to restore -globulin synthesis and reactivate production of fetal hemoglobin. Both patients underwent busulfan-induced myeloablation prior to receiving the treatment.

The investigators suggested that the CRISPR-Cas9-based gene-edited product could change the paradigm for patients with these conditions, if it was found to successfully and durably graft, produce no "off-target" editing products, and, importantly, improve clinical course.

"Recently approved therapies, including luspatercept and crizanlizumab, have reduced transfusion requirements in patients with TDT and the incidence of vaso-occlusive episodes in those with SCD, respectively, but neither treatment addressed the underlying cause of the disease nor fully ameliorates disease manifestations," Frangoul and colleagues wrote.

The investigators reported that both patients had "early, substantial, and sustained increases" in pancellularly distributed fetal hemoglobin levels during the 12-month study period. Further, the patients no longer required transfusions, and the patient with SCD no longer experienced vaso-occlusive episodes after the treatment.

In commentary accompanying the report, Harry Malech, MD, Genetic Immunotherapy Section, Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Disease (NIAID), National Institutes of Health (NIH), Bethesda, MD, described the investigators' application of the gene-editing technology as a "remarkable level of functional correction of the disease phenotype."

"With tangible results for their patients, Frangoul et al have provided a proof of principle of the emerging clinical potential for gene-editing treatments to ameliorate the burden of human disease," Malech pronounced.

In the other published trial, with 6 patients with SCD, Erica Esrick MD, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School, and colleagues described results with infusion of gene-modified cells derived from lentivirus insertion of a gene that knocks down BCL11a by encoding an erythroid-specific, inhibitory short-hairpin RNA (shRNA).

The severity of SCD that qualified patients for enrollment included history of stroke (n = 3), frequent vaso-occlusive events (n = 2) and frequent episodes of priapism (1).Patients were followed for 2 years, and offered enrollment in a 13-year long-term follow-up study.The infusion of the experimental drug BCH-BB694, from the short hairpin RNA embedded within an endogeonous micro RNA scaffold (termed a shmiR vector), was initiated after myeloablation with busulfan.

Esrick and colleagues reported that, at median follow-up of 18 months (range, 7-29), all patients had engraftment and a robust and stable HbF induction broadly distributed in red cells.Clinical manifestations of SCD were reduced or absent during the follow-up period; with no patient having a vaso-occlusive crisis, acute chest syndrome, or stoke subsequent to the gene therapy infusion.Adverse events were consistent with effects of the preparative chemotherapy.

"The field of autologous gene therapies for hemoglobinopathies is advancing rapidly," Esrick and colleagues reported, "including lentiviral trials of gene addition in which the nonsickling hemoglobin is formed from an exogenous -globin or modified -globin gene."

Walters agreed that gene therapy is rapidly progressing, but expressed concern about the large gap that looms between laboratory bench and clinical bedside, particularly for this affected population.

"Access to and delivery of these highly technical therapies in patients with sickle cell disease will be challenging and probably limited to resource-rich nations, at least in the short term," Walters commented.

The studies, CRISPR-Cas9 Gene Editing for Sickle Cell Disease and -Thalassemia, as well as, Post-Transcriptional Genetic Silencing of BCL11A to Treat Sickle Cell Disease, were published online in The New England Journal of Medicine.

Read the original here:
Two Gene Therapies Fix Fault in Sickle Cell Disease and -thalassemia - MD Magazine

Crispr And Crispr-Associated (Cas) Genes Marketresearch report is the new statistical data source added byA2Z Market Research.

Crispr And Crispr-Associated (Cas) Genes Market is growing at a High CAGR during the forecast period 2021-2027. The increasing interest of the individuals in this industry is that the major reason for the expansion of this market.

Crispr And Crispr-Associated (Cas) Genes Marketresearch is an intelligence report with meticulous efforts undertaken to study the right and valuable information. The data which has been looked upon is done considering both, the existing top players and the upcoming competitors. Business strategies of the key players and the new entering market industries are studied in detail. Well explained SWOT analysis, revenue share and contact information are shared in this report analysis.

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Note In order to provide more accurate market forecast, all our reports will be updated before delivery by considering the impact of COVID-19.

Top Key Players Profiled in this report are:

Intellia Therapeutics, GE Healthcare Dharmacon, Caribou Biosciences, Merck KGaA, Editas Medicine, Mirus Bio LLC, Thermo Fisher Scientific, Takara Bio USA, Horizon Discovery Group, CRISPR THERAPEUTICS, Addgene.

The key questions answered in this report:

Various factors are responsible for the markets growth trajectory, which are studied at length in the report. In addition, the report lists down the restraints that are posing threat to the global Crispr And Crispr-Associated (Cas) Genes market. It also gauges the bargaining power of suppliers and buyers, threat from new entrants and product substitute, and the degree of competition prevailing in the market. The influence of the latest government guidelines is also analyzed in detail in the report. It studies the Crispr And Crispr-Associated (Cas) Genes markets trajectory between forecast periods.

Global Crispr And Crispr-Associated (Cas) Genes Market Segmentation:

Market Segmentation: By Type

Genome EditingGenetic engineeringgRNA Database/Gene LibrarCRISPR PlasmidHuman Stem Cells

Market Segmentation: By Application

Biotechnology CompaniesPharmaceutical CompaniesAcademic InstitutesResearch and Development Institutes

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Regions Covered in the Global Crispr And Crispr-Associated (Cas) Genes Market Report 2021:The Middle East and Africa(GCC Countries and Egypt)North America(the United States, Mexico, and Canada)South America(Brazil etc.)Europe(Turkey, Germany, Russia UK, Italy, France, etc.)Asia-Pacific(Vietnam, China, Malaysia, Japan, Philippines, Korea, Thailand, India, Indonesia, and Australia)

The cost analysis of the Global Crispr And Crispr-Associated (Cas) Genes Market has been performed while keeping in view manufacturing expenses, labor cost, and raw materials and their market concentration rate, suppliers, and price trend. Other factors such as Supply chain, downstream buyers, and sourcing strategy have been assessed to provide a complete and in-depth view of the market. Buyers of the report will also be exposed to a study on market positioning with factors such as target client, brand strategy, and price strategy taken into consideration.

The report provides insights on the following pointers:

Market Penetration:Comprehensive information on the product portfolios of the top players in the Crispr And Crispr-Associated (Cas) Genes market.

Product Development/Innovation:Detailed insights on the upcoming technologies, R&D activities, and product launches in the market.

Competitive Assessment: In-depth assessment of the market strategies, geographic and business segments of the leading players in the market.

Market Development:Comprehensive information about emerging markets. This report analyzes the market for various segments across geographies.

Market Diversification:Exhaustive information about new products, untapped geographies, recent developments, and investments in the Crispr And Crispr-Associated (Cas) Genes market.

Table of Contents

Global Crispr And Crispr-Associated (Cas) Genes Market Research Report 2021 2027

Chapter 1 Crispr And Crispr-Associated (Cas) Genes Market Overview

Chapter 2 Global Economic Impact on Industry

Chapter 3 Global Market Competition by Manufacturers

Chapter 4 Global Production, Revenue (Value) by Region

Chapter 5 Global Supply (Production), Consumption, Export, Import by Regions

Chapter 6 Global Production, Revenue (Value), Price Trend by Type

Chapter 7 Global Market Analysis by Application

Chapter 8 Manufacturing Cost Analysis

Chapter 9 Industrial Chain, Sourcing Strategy and Downstream Buyers

Chapter 10 Marketing Strategy Analysis, Distributors/Traders

Chapter 11 Market Effect Factors Analysis

Chapter 12 Global Crispr And Crispr-Associated (Cas) Genes Market Forecast

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Comprehensive Report on Crispr And Crispr-Associated (Cas) Genes Market 2021 | Trends, Growth Demand, Opportunities & Forecast To 2027 | Intellia...

The CRISPR systems inside bacteria serve as adaptive immune systems, but they also threaten to unleash autoimmune reactions. Fortunately, for bacteria such as Streptococcus pyogenes, these systems have a built-in safety feature: a long-form transactivating CRISPR RNA (tracrRNA). Unlike the short-form tracrRNA, which together with CRISPR RNA (crRNA) complexes with the CRISPR-Cas9 enzyme and guides it to DNA sites where it executes cuts, the long-form tracrRNA guides the enzyme to the enzymes own genetic promoter.

The long-form tracrRNA that complexes CRISPR-Cas9 enzyme doesnt need to bind to crRNA, and it doesnt cut. Instead, it merely lingers in place, preventing gene expression.

Essentially, long-form tracrRNA acts as a safety feature, dialing down a bacteriums immune system to prevent it from attacking the bacterium itself rather than foreign DNA. This self-protection function for long-form tracrRNA was uncovered by researchers at Johns Hopkins University. The researchers, led by Joshua W. Modell, PhD, also explored whether long-form tracrRNA could be reprogrammed to guide CRISPR-Cas9 to DNA sites other than the CRISPR-Cas9 promoter.

The researchers findings appeared in the journal Cell, in an article titled, A natural single-guide RNA repurposes Cas9 to autoregulate CRISPR-Cas expression. According to the researchers, long-form tracrRNA could serve as a programmable genetic dimmer switch, one that could be used to inhibit the expression of designated genes in research applications.

We show that in the S. pyogenes CRISPR-Cas system, a long-form transactivating CRISPR RNA folds into a natural single guide that directs Cas9 to transcriptionally repress its own promoter (Pcas), the articles authors wrote. Further, we demonstrate that Pcas serves as a critical regulatory node.

Scientists have long worked to unravel the precise steps of CRISPR-Cas9s mechanism and how its activity in bacteria is dialed up or down. Looking for genes that ignite or inhibit the CRISPR-Cas9 gene-cutting system for the common, strep-throat causing bacterium S. pyogenes, the Johns Hopkins scientists found a clue regarding how that aspect of the system works.

Specifically, the scientists found a gene in the CRISPR-Cas9 system that, when deactivated, led to a dramatic increase in the activity of the system in bacteria. The product of this gene appeared to re-program Cas9 to act as a brake, rather than as a scissor, to dial down the CRISPR system.

From an immunity perspective, bacteria need to ramp up CRISPR-Cas9 activity to identify and rid the cell of threats, but they also need to dial it down to avoid autoimmunitywhen the immune system mistakenly attacks components of the bacteria themselves, said graduate student Rachael Workman, a bacteriologist working in Modells laboratory.

To further nail down the particulars of the brake, the teams next step was to better understand the product of the deactivated gene, a tracrRNA. tracrRNAs belong to a unique family of RNAs that do not make proteins. Instead, they act as a kind of scaffold that allows the Cas9 enzyme to carry the guide RNA that contains a mug shot of previously encountered phage DNA. The mug shot allows Cas9 to cut matching DNA sequences in newly invading viruses.

tracrRNA comes in two sizes: long and short. Most of the modern gene-cutting CRISPR-Cas9 tools use the short form. However, the research team found that the deactivated gene product was the long-form of tracrRNA, the function of which has been entirely unknown.

In bacteria, DNA-cutting CRISPR-Cas9 complexes typically consist of a Cas9 enzyme and a guide RNA. The guide RNA consists of a short-form transactivating CRISPR RNA (tracrRNA) scaffold and a DNA-sequence-specific CRISPR (crRNA). Long-form tracrRNA, however, can complex with and guide Cas9 without crRNA. When long-form tracrRNA does so, it guides the Cas9 enzyme to a Cas9 promoter. The promoter is not cut, but expression is repressed. Left: A schematic of the long-form of the tracrRNA used by the CRISPR-Cas9 system in bacteria. Right: the standard guide RNA used by many scientists as part of the gene-cutting CRISPR-Cas9 system. (Often, the guide RNA is a single synthetic molecule, rather than a combination of tracrRNA and crRNA.) [Joshua Modell and Rachael Workman, Johns Hopkins Medicine]The long and short forms of tracrRNA are similar in structure and have in common the ability to bind to Cas9. The short-form tracrRNA also binds to the guide RNA. However, the long-form tracrRNA doesnt need to bind to the crRNA, because it contains a segment that mimics the crRNA. Essentially, long-form tracrRNAs have combined the function of the short-form tracrRNA and crRNA, explained Modell, assistant professor of molecular biology and genetics at the Johns Hopkins University School of Medicine.

The researchers used genetic engineering to alter the length of a certain region in long-form tracrRNA to make the tracrRNA appear more like a guide RNA. They found that with the altered long-form tracrRNA, Cas9 once again behaved more like a scissor.

Other experiments showed that in lab-grown bacteria with a plentiful amount of long-form tracrRNA, levels of all CRISPR-related genes were very low. When the long-form tracrRNA was removed from bacteria, however, expression of CRISPR-Cas9 genes increased a hundredfold.

Bacterial cells lacking the long-form tracrRNA were cultured in the laboratory for three days and compared with similarly cultured cells containing the long-form tracrRNA. By the end of the experiment, bacteria without the long-form tracrRNA had completely died off. De-repression causes a dramatic 3,000-fold increase in immunization rates against viruses, the articles authors noted. However, heightened immunity comes at the cost of increased autoimmune toxicity.

These findings suggest that long-form tracrRNA normally protects cells from the sickness and death that happen when CRISPR-Cas9 activity is very high. We started to get the idea that the long form was repressing but not eliminating its own CRISPR-related activity, recalled Workman.

To see if the long-form tracrRNA could be re-programmed to repress other bacterial genes, the research team altered the long-form tracrRNAs spacer region to let it sit on a gene that produces green fluorescence. Bacteria with this mutated version of long-form tracrRNA glowed less green than bacteria containing the normal long-form tracrRNA, suggesting that the long-form tracrRNA can be genetically engineered to dial down other bacterial genes.

Another research team, from Emory University, found that in the parasitic bacteria Francisella novicida, Cas9 behaves as a dimmer switch for a gene outside the CRISPR-Cas9 region. The CRISPR-Cas9 system in the Johns Hopkins study is more widely used by scientists as a gene-cutting tool, and the Johns Hopkins teams findings provide evidence that the dimmer action controls the CRISPR-Cas9 system in addition to other genes.

Using bioinformatic analyses, we provide evidence that tracrRNA-mediated autoregulation is widespread in type II-A CRISPR-Cas systems, the Johns Hopkins scientists added. Collectively, we unveil a new paradigm for the intrinsic regulation of CRISPR-Cas systems by natural single guides, which may facilitate the frequent horizontal transfer of these systems into new hosts that have not yet evolved their own regulatory strategies.

The researchers also found the genetic components of long-form tracrRNA in about 40% of the Streptococcus group of bacteria. Further study of bacterial strains that dont have the long-form tracrRNA, said Workman, will potentially reveal whether their CRISPR-Cas9 systems are intact, and other ways that bacteria may dial back the CRISPR-Cas9 system.

The dimmer capability that the experiments uncovered offers opportunities to design new or better CRISPR-Cas9 tools aimed at regulating gene activity for research purposes. In a gene editing scenario, Modell suggested, a researcher may want to cut a specific gene, in addition to using the long-form tracrRNA to inhibit gene activity.

Excerpt from:
Natural CRISPR's Safety Feature Could Become Genetic Dimmer Switch - Genetic Engineering & Biotechnology News